Maria Grazia Roncarolo

George D. Smith Professor of Stem Cell and Regenerative Medicine and Professor of Medicine (Blood and Marrow Transplantation and Cellular Therapy)

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Additional Languages Spoken:

Italian

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Biography

Maria Grazia Roncarolo, MD is the George D. Smith Professor in Stem Cell and Regenerative Medicine, Professor of Pediatrics and of Medicine, director of the Center for Definitive and Curative Medicine, and co-director of the Institute for Stem Cell Biology and Regenerative Medicine.

Dr. Roncarolo leads efforts to translate scientific discoveries in genetic diseases and regenerative medicine into novel patient therapies, including treatments based on stem cells and gene therapy.

A pediatric immunologist by training, she earned her medical degree at the University of Turin, Italy. She spent her early career in Lyon, France, where she focused on severe inherited metabolic and immune diseases, including severe combined immunodeficiency (SCID), better known as the "bubble boy disease." Dr. Roncarolo was a key member of the team that carried out the first stem cell transplants given before birth to treat these genetic diseases.

While studying inherited immune diseases, Dr. Roncarolo discovered a new class of T cells. These cells, called T regulatory type 1 cells, help maintain immune system homeostasis by preventing autoimmune diseases and assisting the immune system in tolerating transplanted cells and organs. Dr. Roncarolo completed the first clinical trial using T regulatory type 1 cells to prevent severe graft-versus-host disease in leukemia patients receiving blood-forming stem-cell transplants from donors who were not genetic matches.

Dr. Roncarolo worked for several years at DNAX Research Institute for Molecular and Cellular Biology in Palo Alto, where she contributed to the discovery of novel cytokines, cell-signaling molecules that are part of the immune response. She studied the role of cytokines in inducing immunological tolerance and in promoting stem cell growth and differentiation.

Dr. Roncarolo developed new gene-therapy approaches, which she pursued as director of the Telethon Institute for Cell and Gene Therapy at the San Raffaele Scientific Institute in Milan. She was the principal investigator leading the successful gene therapy trial for SCID patients who lack an enzyme critical to DNA synthesis, which is a severe life-threatening disorder. Based on the results of this trial, gene therapy for ADA-SCID has obtained Orphan drug status from both the FDA and EMEA and it was licensed to Glaxo Smith Klein, which has received European Commission approval to market under the name of Strimvelis. Under her direction, the San Raffaele Scientific Institute has been seminal in showing the efficacy of gene therapy for otherwise untreatable inherited metabolic diseases and primary immunodeficiencies.

Dr. Roncarolo established the Stanford Center for Definitive and Curative Medicine to cure patients with currently incurable diseases through the development of innovative stem cell-and gene-based therapies.

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Professional Summary

Education & Certifications

Board Certification: Provincial Association of Surgeons and Dentists of Turin, Allergy and Immunology (1987)
Board Certification: Provincial Association of Surgeons and Dentists of Turin, Pediatrics (1983)
Fellowship: University of Milan School of Medicine (1990) Italy
- Residency: University of Turin Children Hospital, Italy (1986) Italy
- Medical Education: University of Turin (1982) Italy
- M.D., University of Turin, Italy, Medicine (1982)
- Natl. Board, University of Turin, Italy, Pediatrics (1986)
- Natl. Board, University of Milan, Italy, Clinical Immunology (1990)

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Honors & Awards

"Gold Apple" Prize for outstanding contribution to science, Marisa Bellisario Foundation (2013)
Elected Member, Academia Europaea of Sciences (2005)
Elected Member, Austrian Academy of Sciences (2012)
- Eurordis Scientific Award 2012 for outstanding contributions to the cure of genetic diseases, Eurordis (2012)
- Knighthood "Commendatore dell'Ordine Al Merito della Repubblica Italiana", President of Italy (2014)
- Nominated "Ufficiale dell'Ordine Al Merito della Repubblica Italiana", President of Italy (2000)
- Outstanding Achievement Award, American Society of Gene & Cell Therapy (2017)
- Outstanding Achievement Award for career and pioneering contributions to the field, European Society of Gene and Cell Therapy (2010)

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Administrative Appointments

Co-Director, Bass Center for Childhood Cancer and Blood Diseases, Lucile Packard Children's Hospital Stanford (2014 - 2019)
Co-Director, Institute for Stem Cell Biology and Regenerative Medicine, Stanford School of Medicine (2014 - Present)
Director, Stanford Center for Definitive and Curative Medicine (CDCM) (2016 - Present)
- Division Chief, Pediatric Stem Cell Transplantation and Regenerative Medicine, Stanford School of Medicine (2014 - 2019)
- Professor, Departments of Pediatrics and Medicine, Stanford University (2014 - Present)

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Publications

Human insulin production and amelioration of diabetes in mice by electrotransfer-enhanced plasmid DNA gene transfer to the skeletal muscle
Martinenghi, S., De Angelis, G. C., Biressi, S., Amadio, S., Bifari, F., Roncarolo, M. G., … Falqui, L. (2002). Human insulin production and amelioration of diabetes in mice by electrotransfer-enhanced plasmid DNA gene transfer to the skeletal muscle. GENE THERAPY, 9(21), 1429–1437.
ISOLATION AND EXPRESSION OF HUMAN CYTOKINE SYNTHESIS INHIBITORY FACTOR CDNA CLONES - HOMOLOGY TO EPSTEIN-BARR-VIRUS OPEN READING FRAME BCRFI
Vieira, P., DEWAALMALEFYT, R., Dang, M. N., Johnson, K. E., Kastelein, R., Fiorentino, D. F., … Moore, K. W. (1991). ISOLATION AND EXPRESSION OF HUMAN CYTOKINE SYNTHESIS INHIBITORY FACTOR CDNA CLONES - HOMOLOGY TO EPSTEIN-BARR-VIRUS OPEN READING FRAME BCRFI. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 88(4), 1172–1176.
IL-10 IS PRODUCED BY SUBSETS OF HUMAN CD4+ T-CELL CLONES AND PERIPHERAL-BLOOD T-CELLS
Yssel, H., Malefyt, R. D., Roncarolo, M. G., Abrams, J. S., Lahesmaa, R., Spits, H., & deVries, J. E. (1992). IL-10 IS PRODUCED BY SUBSETS OF HUMAN CD4+ T-CELL CLONES AND PERIPHERAL-BLOOD T-CELLS. JOURNAL OF IMMUNOLOGY, 149(7), 2378–2384.
- Molecular purging of multiple myeloma cells by ex-vivo culture and retroviral transduction of mobilized-blood CD34(+) cells
  Deola, S., Scaramuzza, S., Birolo, R. S., Cergnul, M., Ficara, F., Dando, J., … Bregni, M. (2007). Molecular purging of multiple myeloma cells by ex-vivo culture and retroviral transduction of mobilized-blood CD34(+) cells. JOURNAL OF TRANSLATIONAL MEDICINE, 5.
- Evidence for Long-term Efficacy and Safety of Gene Therapy for Wiskott-Aldrich Syndrome in Preclinical Models
  Marangoni, F., Bosticardo, M., Charrier, S., Draghici, E., Locci, M., Scaramuzza, S., … Villa, A. (2009). Evidence for Long-term Efficacy and Safety of Gene Therapy for Wiskott-Aldrich Syndrome in Preclinical Models. MOLECULAR THERAPY, 17(6), 1073–1082.
- WASP regulates suppressor activity of human and murine CD4(+)CD25(+)FOXP3(+) natural regulatory T cells
  Marangoni, F., Trifari, S., Scaramuzza, S., Panaroni, C., Martino, S., Notarangelo, L. D., … Dupre, L. (2007). WASP regulates suppressor activity of human and murine CD4(+)CD25(+)FOXP3(+) natural regulatory T cells. JOURNAL OF EXPERIMENTAL MEDICINE, 204(2), 369–380.
- Tr1 cells and the counter-regulation of immunity: natural mechanisms and therapeutic applications.
  Roncarolo, M. G., Gregori, S., Bacchetta, R., & Battaglia, M. (2014). Tr1 cells and the counter-regulation of immunity: natural mechanisms and therapeutic applications. Current Topics in Microbiology and Immunology, 380, 39–68.
- Forkhead box P3: The Peacekeeper of the Immune System
  Passerini, L., de Sio, F. R. S., Roncarolo, M. G., & Bacchetta, R. (2014). Forkhead box P3: The Peacekeeper of the Immune System. INTERNATIONAL REVIEWS OF IMMUNOLOGY, 33(2), 129–145.
- CD4(+) T Cells from IPEX Patients Convert into Functional and Stable Regulatory T Cells by FOXP3 Gene Transfer
  Passerini, L., Mel, E. R., Sartirana, C., Fousteri, G., Bondanza, A., Naldini, L., … Bacchetta, R. (2013). CD4(+) T Cells from IPEX Patients Convert into Functional and Stable Regulatory T Cells by FOXP3 Gene Transfer. SCIENCE TRANSLATIONAL MEDICINE, 5(215).
- Coexpression of CD49b and LAG-3 identifies human and mouse T regulatory type 1 cells
  Gagliani, N., Magnani, C. F., Huber, S., Gianolini, M. E., Pala, M., Licona-Limon, P., … Roncarolo, M.-G. (2013). Coexpression of CD49b and LAG-3 identifies human and mouse T regulatory type 1 cells. NATURE MEDICINE, 19(6), 739-?
- Human IL2RA null mutation mediates immunodeficiency with lymphoproliferation and autoimmunity
  Goudy, K., Aydin, D., Barzaghi, F., Gambineri, E., Vignoli, M., Mannurita, S. C., … Bacchetta, R. (2013). Human IL2RA null mutation mediates immunodeficiency with lymphoproliferation and autoimmunity. CLINICAL IMMUNOLOGY, 146(3), 248–261.
- BAT2 and BAT3 polymorphisms as novel genetic risk factors for rejection after HLA-related SCT
  Piras, I. S., Angius, A., Andreani, M., Testi, M., Lucarelli, G., Floris, M., … Bacchetta, R. (2014). BAT2 and BAT3 polymorphisms as novel genetic risk factors for rejection after HLA-related SCT. BONE MARROW TRANSPLANTATION, 49(11), 1400–1404.
- Sirolimus-based graft-versus-host disease prophylaxis promotes the in vivo expansion of regulatory T cells and permits peripheral blood stem cell transplantation from haploidentical donors
  Peccatori, J., Forcina, A., Clerici, D., Crocchiolo, R., Vago, L., Stanghellini, M. T. L., … Ciceri, F. (2015). Sirolimus-based graft-versus-host disease prophylaxis promotes the in vivo expansion of regulatory T cells and permits peripheral blood stem cell transplantation from haploidentical donors. LEUKEMIA, 29(2), 396–405.
- B-cell development and functions and therapeutic options in adenosine deaminase-deficient patients
  Brigida, I., Sauer, A. V., Ferrua, F., Giannelli, S., Scaramuzza, S., Pistoia, V., … Aiuti, A. (2014). B-cell development and functions and therapeutic options in adenosine deaminase-deficient patients. JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY, 133(3), 799-?
- Liver gene therapy by lentiviral vectors reverses anti-factor IX pre-existing immunity in haemophilic mice
  Annoni, A., Cantore, A., Della Valle, P., Goudy, K., Akbarpour, M., Russo, F., … Naldini, L. (2013). Liver gene therapy by lentiviral vectors reverses anti-factor IX pre-existing immunity in haemophilic mice. EMBO MOLECULAR MEDICINE, 5(11), 1684–1697.
- Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy.
  Biffi, A., Montini, E., Lorioli, L., Cesani, M., Fumagalli, F., Plati, T., … Naldini, L. (2013). Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science , 341(6148), 1233158-?
- Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome.
  Aiuti, A., Biasco, L., Scaramuzza, S., Ferrua, F., Cicalese, M. P., Baricordi, C., … Naldini, L. (2013). Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science , 341(6148), 1233151-?
- Transplant Tolerance to Pancreatic Islets Is Initiated in the Graft and Sustained in the Spleen
  Gagliani, N., Jofra, T., Valle, A., Stabilini, A., Morsiani, C., Gregori, S., … Battaglia, M. (2013). Transplant Tolerance to Pancreatic Islets Is Initiated in the Graft and Sustained in the Spleen. AMERICAN JOURNAL OF TRANSPLANTATION, 13(8), 1963–1975.
- Regulatory T cells: recommendations to simplify the nomenclature.
  Abbas, A. K., Benoist, C., Bluestone, J. A., Campbell, D. J., Ghosh, S., Hori, S., … Ziegler, S. F. (2013). Regulatory T cells: recommendations to simplify the nomenclature. Nature Immunology, 14(4), 307–308.
- Immune responses in liver-directed lentiviral gene therapy
  Annoni, A., Goudy, K., Akbarpour, M., Naldini, L., & Roncarolo, M. G. (2013). Immune responses in liver-directed lentiviral gene therapy. TRANSLATIONAL RESEARCH, 161(4), 230–240.
- HLA-G expressing DC-10 and CD4(+) T cells accumulate in human decidua during pregnancy
  Amodio, G., Mugione, A., Sanchez, A. M., Vigano, P., Candiani, M., Somigliana, E., … Gregori, S. (2013). HLA-G expressing DC-10 and CD4(+) T cells accumulate in human decidua during pregnancy. HUMAN IMMUNOLOGY, 74(4), 406–411.
- A novel function for FOXP3 in humans: intrinsic regulation of conventional T cells
  McMurchy, A. N., Gillies, J., Gizzi, M. C., Riba, M., Garcia-Manteiga, J. M., Cittaro, D., … Levings, M. K. (2013). A novel function for FOXP3 in humans: intrinsic regulation of conventional T cells. BLOOD, 121(8), 1265–1275.
- Health related quality of life in Middle Eastern children with beta-thalassemia.
  Caocci, G., Efficace, F., Ciotti, F., Roncarolo, M. G., Vacca, A., Piras, E., … La Nasa, G. (2012). Health related quality of life in Middle Eastern children with beta-thalassemia. BMC Blood Disorders, 12, 6-?
- Enforced IL-10 Expression Confers Type 1 Regulatory T Cell (Tr1) Phenotype and Function to Human CD4(+) T Cells
  Andolfi, G., Fousteri, G., Rossetti, M., Magnani, C. F., Jofra, T., Locafaro, G., … Roncarolo, M.-G. (2012). Enforced IL-10 Expression Confers Type 1 Regulatory T Cell (Tr1) Phenotype and Function to Human CD4(+) T Cells. MOLECULAR THERAPY, 20(9), 1778–1790.
- The cellular and molecular mechanisms of immuno-suppression by human type 1 regulatory T cells.
  Gregori, S., Goudy, K. S., & Roncarolo, M. G. (2012). The cellular and molecular mechanisms of immuno-suppression by human type 1 regulatory T cells. Frontiers in Immunology, 3, 30-?
- Genotypes and haplotypes in the 3' untranslated region of the HLA-G gene and their association with clinical outcome of hematopoietic stem cell transplantation for beta-thalassemia
  Sizzano, F., Testi, M., Zito, L., Crocchiolo, R., Troiano, M., Mazzi, B., … Fleischhauer, K. (2012). Genotypes and haplotypes in the 3' untranslated region of the HLA-G gene and their association with clinical outcome of hematopoietic stem cell transplantation for beta-thalassemia. TISSUE ANTIGENS, 79(5), 326–332.
- Preclinical Safety and Efficacy of Human CD34(+) Cells Transduced With Lentiviral Vector for the Treatment of Wiskott-Aldrich Syndrome
  Scaramuzza, S., Biasco, L., Ripamonti, A., Castiello, M. C., Loperfido, M., Draghici, E., … Aiuti, A. (2013). Preclinical Safety and Efficacy of Human CD34(+) Cells Transduced With Lentiviral Vector for the Treatment of Wiskott-Aldrich Syndrome. MOLECULAR THERAPY, 21(1), 175–184.
- Demethylation analysis of the FOXP3 locus shows quantitative defects of regulatory T cells in IPEX-like syndrome
  Barzaghi, F., Passerini, L., Gambineri, E., Mannurita, S. C., Cornu, T., Kang, E. S., … Bacchetta, R. (2012). Demethylation analysis of the FOXP3 locus shows quantitative defects of regulatory T cells in IPEX-like syndrome. JOURNAL OF AUTOIMMUNITY, 38(1), 49–58.
- Dendritic cell functional improvement in a preclinical model of lentiviral-mediated gene therapy for Wiskott-Aldrich syndrome
  Catucci, M., Prete, F., Bosticardo, M., Castiello, M. C., Draghici, E., LOCCI, M., … Villa, A. (2012). Dendritic cell functional improvement in a preclinical model of lentiviral-mediated gene therapy for Wiskott-Aldrich syndrome. GENE THERAPY, 19(12), 1150–1158.
- Alterations in the adenosine metabolism and CD39/CD73 adenosinergic machinery cause loss of Treg cell function and autoimmunity in ADA-deficient SCID
  Sauer, A. V., Brigida, I., Carriglio, N., Hernandez, R. J., Scaramuzza, S., Clavenna, D., … Aiuti, A. (2012). Alterations in the adenosine metabolism and CD39/CD73 adenosinergic machinery cause loss of Treg cell function and autoimmunity in ADA-deficient SCID. BLOOD, 119(6), 1428–1439.
- Rapamycin Combined with Anti-CD45RB mAb and IL-10 or with G-CSF Induces Tolerance in a Stringent Mouse Model of Islet Transplantation
  Gagliani, N., Gregori, S., Jofra, T., Valle, A., Stabilini, A., Rothstein, D. M., … Battaglia, M. (2011). Rapamycin Combined with Anti-CD45RB mAb and IL-10 or with G-CSF Induces Tolerance in a Stringent Mouse Model of Islet Transplantation. PLOS ONE, 6(12).
- Manipulating Immune Tolerance with Micro-RNA Regulated Gene Therapy.
  Goudy, K. S., Annoni, A., Naldini, L., & Roncarolo, M.-G. (2011). Manipulating Immune Tolerance with Micro-RNA Regulated Gene Therapy. Frontiers in Microbiology, 2, 221-?
- Forkhead box protein 3 (FOXP3) mutations lead to increased T(H)17 cell numbers and regulatory T-cell instability
  Passerini, L., Olek, S., Di Nunzio, S., Barzaghi, F., Hambleton, S., Abinun, M., … Bacchetta, R. (2011). Forkhead box protein 3 (FOXP3) mutations lead to increased T(H)17 cell numbers and regulatory T-cell instability. JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY, 128(6), 1376–U790.
- Immune intervention with T regulatory cells: Past lessons and future perspectives for type 1 diabetes
  Battaglia, M., & Roncarolo, M.-G. (2011). Immune intervention with T regulatory cells: Past lessons and future perspectives for type 1 diabetes. SEMINARS IN IMMUNOLOGY, 23(3), 182–194.
- Stability of human rapamycin-expanded CD4(+)CD25(+) T regulatory cells
  Tresoldi, E., Dell'albani, I., Stabilini, A., Jofra, T., Valle, A., Gagliani, N., … Battaglia, M. (2011). Stability of human rapamycin-expanded CD4(+)CD25(+) T regulatory cells. HAEMATOLOGICA-THE HEMATOLOGY JOURNAL, 96(9), 1357–1365.
- Lentiviral-mediated gene therapy leads to improvement of B-cell functionality in a murine model of Wiskott-Aldrich syndrome
  Bosticardo, M., Draghici, E., Schena, F., Sauer, A. V., Fontana, E., Castiello, M. C., … Villa, A. (2011). Lentiviral-mediated gene therapy leads to improvement of B-cell functionality in a murine model of Wiskott-Aldrich syndrome. JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY, 127(6), 1376–U109.
- Hepatocyte-Targeted Expression by Integrase-Defective Lentiviral Vectors Induces Antigen-Specific Tolerance in Mice with Low Genotoxic Risk
  Matrai, J., Cantore, A., Bartholomae, C. C., Annoni, A., Wang, W., Acosta-Sanchez, A., … Naldini, L. (2011). Hepatocyte-Targeted Expression by Integrase-Defective Lentiviral Vectors Induces Antigen-Specific Tolerance in Mice with Low Genotoxic Risk. HEPATOLOGY, 53(5), 1696–1707.
- Th17 Cells Express Interleukin-10 Receptor and Are Controlled by Foxp3(-) and Foxp3(+) Regulatory CD4(+) T Cells in an Interleukin-10-Dependent Manner
  Huber, S., Gagliani, N., Esplugues, E., O'Connor, W., Huber, F. J., Chaudhry, A., … Flavell, R. A. (2011). Th17 Cells Express Interleukin-10 Receptor and Are Controlled by Foxp3(-) and Foxp3(+) Regulatory CD4(+) T Cells in an Interleukin-10-Dependent Manner. IMMUNITY, 34(4), 554–565.
- Clinical tolerance in allogeneic hematopoietic stem cell transplantation
  Roncarolo, M.-G., Gregori, S., Lucarelli, B., Ciceri, F., & Bacchetta, R. (2011). Clinical tolerance in allogeneic hematopoietic stem cell transplantation. IMMUNOLOGICAL REVIEWS, 241, 145–163.
- Killing of myeloid APCs via HLA class I, CD2 and CD226 defines a novel mechanism of suppression by human Tr1 cells
  Magnani, C. F., Alberigo, G., Bacchetta, R., Serafini, G., Andreani, M., Roncarolo, M. G., & Gregori, S. (2011). Killing of myeloid APCs via HLA class I, CD2 and CD226 defines a novel mechanism of suppression by human Tr1 cells. EUROPEAN JOURNAL OF IMMUNOLOGY, 41(6), 1652–1662.
- Functional type 1 regulatory T cells develop regardless of FOXP3 mutations in patients with IPEX syndrome
  Passerini, L., Di Nunzio, S., Gregori, S., Gambineri, E., Cecconi, M., Seidel, M. G., … Bacchetta, R. (2011). Functional type 1 regulatory T cells develop regardless of FOXP3 mutations in patients with IPEX syndrome. EUROPEAN JOURNAL OF IMMUNOLOGY, 41(4), 1120–1131.
- Integration profile of retroviral vector in gene therapy treated patients is cell-specific according to gene expression and chromatin conformation of target cell
  Biasco, L., Ambrosi, A., Pellin, D., Bartholomae, C., Brigida, I., Roncarolo, M. G., … Aiuti, A. (2011). Integration profile of retroviral vector in gene therapy treated patients is cell-specific according to gene expression and chromatin conformation of target cell. EMBO MOLECULAR MEDICINE, 3(2), 89–101.
- Point mutants of forkhead box P3 that cause immune dysregulation, polyendocrinopathy, enteropathy, X-linked have diverse abilities to reprogram T cells into regulatory T cells
  McMurchy, A. N., Gillies, J., Allan, S. E., Passerini, L., Gambineri, E., Roncarolo, M. G., … Levings, M. K. (2010). Point mutants of forkhead box P3 that cause immune dysregulation, polyendocrinopathy, enteropathy, X-linked have diverse abilities to reprogram T cells into regulatory T cells. JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY, 126(6), 1242–1251.
- Bone Marrow as a Source of Hematopoietic Stem Cells for Human Gene Therapy of beta-Thalassemia
  Frittoli, M. C., Biral, E., Cappelli, B., Zambelli, M., Roncarolo, M. G., Ferrari, G., … Marktel, S. (2011). Bone Marrow as a Source of Hematopoietic Stem Cells for Human Gene Therapy of beta-Thalassemia. HUMAN GENE THERAPY, 22(4), 507–513.
- Granulocyte-colony stimulating factor drives the in vitro differentiation of human dendritic cells that induce anergy in naive T cells
  Rossetti, M., Gregori, S., & Roncarolo, M. G. (2010). Granulocyte-colony stimulating factor drives the in vitro differentiation of human dendritic cells that induce anergy in naive T cells. EUROPEAN JOURNAL OF IMMUNOLOGY, 40(11), 3097–3106.
- Methods for in vitro generation of human type 1 regulatory T cells.
  Gregori, S., Roncarolo, M. G., & Bacchetta, R. (2011). Methods for in vitro generation of human type 1 regulatory T cells. Methods in Molecular Biology (Clifton, N.J.), 677, 31–46.
- Prospective Assessment of Health-Related Quality of Life in Pediatric Patients with Beta-Thalassemia following Hematopoietic Stem Cell Transplantation
  Caocci, G., Efficace, F., Ciotti, F., Roncarolo, M. G., Vacca, A., Piras, E., … La Nasa, G. (2011). Prospective Assessment of Health-Related Quality of Life in Pediatric Patients with Beta-Thalassemia following Hematopoietic Stem Cell Transplantation. BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 17(6), 861–866.
- HIV-1-Derived Lentiviral Vectors Directly Activate Plasmacytoid Dendritic Cells, Which in Turn Induce the Maturation of Myeloid Dendritic Cells
  Rossetti, M., Gregori, S., Hauben, E., Brown, B. D., Sergi, L. S., Naldini, L., & Roncarolo, M.-G. (2011). HIV-1-Derived Lentiviral Vectors Directly Activate Plasmacytoid Dendritic Cells, Which in Turn Induce the Maturation of Myeloid Dendritic Cells. HUMAN GENE THERAPY, 22(2), 177–188.
- Molecular and functional characterization of allogantigen-specific anergic T cells suitable for cell therapy
  Bacchetta, R., Gregori, S., Serafini, G., Sartirana, C., Schulz, U., Zino, E., … Roncarolo, M. G. (2010). Molecular and functional characterization of allogantigen-specific anergic T cells suitable for cell therapy. HAEMATOLOGICA-THE HEMATOLOGY JOURNAL, 95(12), 2134–2143.
- Correction of beta-thalassemia major by gene transfer in haematopoietic progenitors of pediatric patients
  Roselli, E. A., Mezzadra, R., Frittoli, M. C., Maruggi, G., Biral, E., Mavilio, F., … Ferrari, G. (2010). Correction of beta-thalassemia major by gene transfer in haematopoietic progenitors of pediatric patients. EMBO MOLECULAR MEDICINE, 2(8), 315–328.
- Role of reduced intensity conditioning in T-cell and B-cell immune reconstitution after HLA-identical bone marrow transplantation in adenosine-deaminase severe combined immunodeficiency
  Cancrini, C., Ferrua, F., Scarselli, A., Brigida, I., Romiti, M. L., Barera, G., … Aiuti, A. (2010). Role of reduced intensity conditioning in T-cell and B-cell immune reconstitution after HLA-identical bone marrow transplantation in adenosine-deaminase severe combined immunodeficiency. HAEMATOLOGICA-THE HEMATOLOGY JOURNAL, 95(10), 1778–1782.
- Differentiation of type 1 T regulatory cells (Tr1) by tolerogenic DC-10 requires the IL-10-dependent ILT4/HLA-G pathway
  Gregori, S., Tomasoni, D., Pacciani, V., Scirpoli, M., Battaglia, M., Magnani, C. F., … Roncarolo, M.-G. (2010). Differentiation of type 1 T regulatory cells (Tr1) by tolerogenic DC-10 requires the IL-10-dependent ILT4/HLA-G pathway. BLOOD, 116(6), 935–944.
- Fatal vancomycin- and linezolid-resistant Enterococcus faecium sepsis in a child undergoing allogeneic haematopoietic stem cell transplantation for beta-thalassaemia major
  Fossati, M., Cappelli, B., Biral, E., Chiesa, R., Biffi, A., Ossi, C., … Marktel, S. (2010). Fatal vancomycin- and linezolid-resistant Enterococcus faecium sepsis in a child undergoing allogeneic haematopoietic stem cell transplantation for beta-thalassaemia major. JOURNAL OF MEDICAL MICROBIOLOGY, 59(7), 839–842.
- High incidence of severe cyclosporine neurotoxicity in children affected by haemoglobinopaties undergoing myeloablative haematopoietic stem cell transplantation: early diagnosis and prompt intervention ameliorates neurological outcome
  Noe, A., Cappelli, B., Biffi, A., Chiesa, R., Frugnoli, I., Biral, E., … Marktel, S. (2010). High incidence of severe cyclosporine neurotoxicity in children affected by haemoglobinopaties undergoing myeloablative haematopoietic stem cell transplantation: early diagnosis and prompt intervention ameliorates neurological outcome. ITALIAN JOURNAL OF PEDIATRICS, 36.
- Revertant T lymphocytes in a patient with Wiskott-Aldrich syndrome: Analysis of function and distribution in lymphoid organs
  Trifari, S., Scaramuzza, S., Catucci, M., Ponzoni, M., Mollica, L., Chiesa, R., … Dupre, L. (2010). Revertant T lymphocytes in a patient with Wiskott-Aldrich syndrome: Analysis of function and distribution in lymphoid organs. JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY, 125(2), 439–448.
- Induction of anergic allergen-specific suppressor T cells using tolerogenic dendritic cells derived from children with allergies to house dust mites
  Pacciani, V., Gregori, S., Chini, L., Corrente, S., Chianca, M., Moschese, V., … Angelini, F. (2010). Induction of anergic allergen-specific suppressor T cells using tolerogenic dendritic cells derived from children with allergies to house dust mites. JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY, 125(3), 727–736.
- Platelet transfusion refractoriness in highly immunized beta thalassemia children undergoing stem cell transplantation
  Marktel, S., Napolitano, S., Zino, E., Cappelli, B., Chiesa, R., Poli, F., … Fleischhauer, K. (2010). Platelet transfusion refractoriness in highly immunized beta thalassemia children undergoing stem cell transplantation. PEDIATRIC TRANSPLANTATION, 14(3), 393–401.
- Ten years of gene therapy for primary immune deficiencies.
  Aiuti, A., & Roncarolo, M. G. (2009). Ten years of gene therapy for primary immune deficiencies. Hematology / the Education Program of the American Society of Hematology. American Society of Hematology. Education Program, 682–689.
- Unpredictability of Intravenous Busulfan Pharmacokinetics in Children Undergoing Hematopoietic Stem Cell Transplantation for Advanced Beta Thalassemia: Limited Toxicity with a Dose-Adjustment Policy
  Chiesa, R., Cappelli, B., Crocchiolo, R., Frugnoli, I., Biral, E., Noe, A., … Marktel, S. (2010). Unpredictability of Intravenous Busulfan Pharmacokinetics in Children Undergoing Hematopoietic Stem Cell Transplantation for Advanced Beta Thalassemia: Limited Toxicity with a Dose-Adjustment Policy. BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 16(5), 622–628.
- [Gene therapy in pediatrics].
  Aiuti, A., Cappelli, B., Biffi, A., Marktel, S., & Roncarolo, M. G. (2009). [Gene therapy in pediatrics]. Minerva Pediatrica, 61(6), 775–778.
- Antigen-Specific Dependence of Tr1-Cell Therapy in Preclinical Models of Islet Transplant
  Gagliani, N., Jofra, T., Stabilini, A., Valle, A., Atkinson, M., Roncarolo, M.-G., & Battaglia, M. (2010). Antigen-Specific Dependence of Tr1-Cell Therapy in Preclinical Models of Islet Transplant. DIABETES, 59(2), 433–439.
- The Tregs' world according to GARP
  Battaglia, M., & Roncarolo, M. G. (2009). The Tregs' world according to GARP. EUROPEAN JOURNAL OF IMMUNOLOGY, 39(12), 3296–3300.
- Escalating doses of donor lymphocytes for incipient graft rejection following SCT for thalassemia
  Frugnoli, I., Cappelli, B., Chiesa, R., Biral, E., Noe, A., Evangelio, C., … Marktel, S. (2010). Escalating doses of donor lymphocytes for incipient graft rejection following SCT for thalassemia. BONE MARROW TRANSPLANTATION, 45(6), 1047–1051.
- In vivo delivery of a microRNA-regulated transgene induces antigen-specific regulatory T cells and promotes immunologic tolerance
  Annoni, A., Brown, B. D., Cantore, A., Sergi, L. S., Naldini, L., & Roncarolo, M.-G. (2009). In vivo delivery of a microRNA-regulated transgene induces antigen-specific regulatory T cells and promotes immunologic tolerance. BLOOD, 114(25), 5152–5161.
- Absence of VOD in paediatric thalassaemic HSCT recipients using defibrotide prophylaxis and intravenous Busulphan
  Cappelli, B., Chiesa, R., Evangelio, C., Biffi, A., Roccia, T., Frugnoli, I., … Marktel, S. (2009). Absence of VOD in paediatric thalassaemic HSCT recipients using defibrotide prophylaxis and intravenous Busulphan. BRITISH JOURNAL OF HAEMATOLOGY, 147(4), 554–560.
- Wild-type FOXP3 is selectively active in CD4(+)CD25(hi) regulatory T cells of healthy female carriers of different FOXP3 mutations
  Di Nunzio, S., Cecconi, M., Passerini, L., McMurchy, A. N., Baron, U., Turbachova, I., … Bacchetta, R. (2009). Wild-type FOXP3 is selectively active in CD4(+)CD25(hi) regulatory T cells of healthy female carriers of different FOXP3 mutations. BLOOD, 114(19), 4138–4141.
- Role of human leukocyte antigen-G in the induction of adaptive type 1 regulatory T cells
  Gregori, S., Magnani, C. F., & Roncarolo, M.-G. (2009). Role of human leukocyte antigen-G in the induction of adaptive type 1 regulatory T cells. HUMAN IMMUNOLOGY, 70(12), 966–969.
- Integration of retroviral vectors induces minor changes in the transcriptional activity of T cells from ADA-SCID patients treated with gene therapy
  Cassani, B., Montini, E., Maruggi, G., Ambrosi, A., Mirolo, M., Selleri, S., … Aiuti, A. (2009). Integration of retroviral vectors induces minor changes in the transcriptional activity of T cells from ADA-SCID patients treated with gene therapy. BLOOD, 114(17), 3546–3556.
- Autoimmune diabetic patients undergoing allogeneic islet transplantation: are we ready for a regulatory T-cell therapy?
  Gagliani, N., Ferraro, A., Roncarolo, M. G., & Battaglia, M. (2009). Autoimmune diabetic patients undergoing allogeneic islet transplantation: are we ready for a regulatory T-cell therapy? IMMUNOLOGY LETTERS, 127(1), 1–7.
- Loss of Mismatched HLA in Leukemia after Stem-Cell Transplantation.
  Vago, L., Perna, S. K., Zanussi, M., Mazzi, B., Barlassina, C., Stanghellini, M. T. L., … Fleischhauer, K. (2009). Loss of Mismatched HLA in Leukemia after Stem-Cell Transplantation. NEW ENGLAND JOURNAL OF MEDICINE, 361(5), 478–488.
- ADA-deficient SCID is associated with a specific microenvironment and bone phenotype characterized by RANKL/OPG imbalance and osteoblast insufficiency
  Sauer, A. V., Mrak, E., Hernandez, R. J., Zacchi, E., Cavani, F., Casiraghi, M., … Aiuti, A. (2009). ADA-deficient SCID is associated with a specific microenvironment and bone phenotype characterized by RANKL/OPG imbalance and osteoblast insufficiency. BLOOD, 114(15), 3216–3226.
- Type 1 regulatory T cells are associated with persistent split erythroid/lymphoid chimerism after allogeneic hematopoietic stem cell transplantation for thalassemia
  Serafini, G., Andreani, M., Testi, M., Battarra, M., Bontadini, A., Biral, E., … Bacchetta, R. (2009). Type 1 regulatory T cells are associated with persistent split erythroid/lymphoid chimerism after allogeneic hematopoietic stem cell transplantation for thalassemia. HAEMATOLOGICA-THE HEMATOLOGY JOURNAL, 94(10), 1415–1426.
- Characterization of New Arylsulfatase A Gene Mutations Reinforces Genotype-Phenotype Correlation in Metachromatic Leukodystrophy
  Cesani, M., Capotondo, A., Plati, T., Sergi, L. S., Fumagalli, F., Roncarolo, M. G., … Biffi, A. (2009). Characterization of New Arylsulfatase A Gene Mutations Reinforces Genotype-Phenotype Correlation in Metachromatic Leukodystrophy. HUMAN MUTATION, 30(10), E936–E945.
- The Fate of Human Treg Cells
  Battaglia, M., & Roncarolo, M. G. (2009). The Fate of Human Treg Cells. IMMUNITY, 30(6), 763–765.
- The Wiskott-Aldrich syndrome protein is required for iNKT cell maturation and function
  Locci, M., Draghici, E., Marangoni, F., Bosticardo, M., Catucci, M., Aiuti, A., … Villa, A. (2009). The Wiskott-Aldrich syndrome protein is required for iNKT cell maturation and function. JOURNAL OF EXPERIMENTAL MEDICINE, 206(4), 735–742.
- Hematopoietic stem cell gene therapy for adenosine deaminase deficient-SCID
  Aiuti, A., Brigida, I., Ferrua, F., Cappelli, B., Chiesa, R., Marktel, S., & Roncarolo, M.-G. (2009). Hematopoietic stem cell gene therapy for adenosine deaminase deficient-SCID. IMMUNOLOGIC RESEARCH, 44(1-3), 150–159.
- Re-establishing immune tolerance in type 1 diabetes via regulatory T cells.
  Gregori, S., Battaglia, M., & Roncarolo, M.-G. (2008). Re-establishing immune tolerance in type 1 diabetes via regulatory T cells. Novartis Foundation Symposium, 292, 174–183.
- Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency.
  Aiuti, A., Cattaneo, F., Galimberti, S., Benninghoff, U., Cassani, B., Callegaro, L., … Roncarolo, M.-G. (2009). Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency. NEW ENGLAND JOURNAL OF MEDICINE, 360(5), 447–458.
- Rapamycin Prevents and Breaks the Anti-CD3-Induced Tolerance in NOD Mice
  Valle, A., Jofra, T., Stabilini, A., Atkinson, M., Roncarolo, M.-G., & Battaglia, M. (2009). Rapamycin Prevents and Breaks the Anti-CD3-Induced Tolerance in NOD Mice. DIABETES, 58(4), 875–881.
- Molecular Regulation of Cellular Immunity by FOXP3
  McMurchy, A. N., Di Nunzio, S., Roncarolo, M. G., Bacchetta, R., & Levings, M. K. (2009). Molecular Regulation of Cellular Immunity by FOXP3. FORKHEAD TRANSCRIPTION FACTORS: VITAL ELEMENTS IN BIOLOGY AND MEDICINE, 665, 30–46.
- Clinical and molecular profile of a new series of patients with immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome: inconsistent correlation between forkhead box protein 3 expression and disease severity.
  Gambineri, E., Perroni, L., Passerini, L., Bianchi, L., Doglioni, C., Meschi, F., … Bacchetta, R. (2008). Clinical and molecular profile of a new series of patients with immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome: inconsistent correlation between forkhead box protein 3 expression and disease severity. Journal of Allergy and Clinical Immunology, 122(6), 1105–1112 e1.
- Clinical improvement and normalized Th1 cytokine profile in early and long-term interferon-alpha treatment in a suspected case of hyper-IgE syndrome
  Benninghoff, U., Cattaneo, F., Aiuti, A., Flores-D'Arcais, A., Gelmetti, C., Viscardi, M., … Bacchetta, R. (2008). Clinical improvement and normalized Th1 cytokine profile in early and long-term interferon-alpha treatment in a suspected case of hyper-IgE syndrome. PEDIATRIC ALLERGY AND IMMUNOLOGY, 19(6), 564–568.
- Metachromatic leukodystrophy - mutation analysis provides further evidence of genotype-phenotype correlation
  Biffi, A., Cesani, M., Fumagalli, F., Del Carro, U., Baldoli, C., Canale, S., … Sessa, M. (2008). Metachromatic leukodystrophy - mutation analysis provides further evidence of genotype-phenotype correlation. CLINICAL GENETICS, 74(4), 349–357.
- Temporal, quantitative, and functional characteristics of single-KIR-positive alloreactive natural killer cell recovery account for impaired graft-versus-leukemia activity after haploidentical hematopoietic stem cell transplantation
  Vago, L., Forno, B., Sormani, M. P., Crocchiolo, R., Zino, E., Di Terlizzi, S., … Fleischhauer, K. (2008). Temporal, quantitative, and functional characteristics of single-KIR-positive alloreactive natural killer cell recovery account for impaired graft-versus-leukemia activity after haploidentical hematopoietic stem cell transplantation. BLOOD, 112(8), 3488–3499.
- CD4(+) T-regulatory cells: toward therapy for human diseases
  Allan, S. E., Broady, R., Gregori, S., Himmel, M. E., Locke, N., Roncarolo, M. G., … Levings, M. K. (2008). CD4(+) T-regulatory cells: toward therapy for human diseases. IMMUNOLOGICAL REVIEWS, 223, 391–421.
- Second hematopoietic SCT in patients with thalassemia recurrence following rejection of the first graft
  Gaziev, J., Sodani, P., Lucarelli, G., Polchi, P., Marktel, S., Paciaroni, K., … Roncarolo, M. G. (2008). Second hematopoietic SCT in patients with thalassemia recurrence following rejection of the first graft. BONE MARROW TRANSPLANTATION, 42(6), 397–404.
- Multiple BM harvests in pediatric donors for thalassemic siblings: safety, efficacy and ethical issues
  Biral, E., Chiesa, R., Cappelli, B., Roccia, T., Frugnoli, I., Noe, A., … Marktel, S. (2008). Multiple BM harvests in pediatric donors for thalassemic siblings: safety, efficacy and ethical issues. BONE MARROW TRANSPLANTATION, 42(6), 379–384.
- Rapamycin monotherapy in patients with type 1 diabetes modifies CD4(+)CD25(+)FOXP3(+) regulatory T-Cells
  Monti, P., Scirpoli, M., Maffi, P., Piemonti, L., Secchi, A., Bonifacio, E., … Battaglia, M. (2008). Rapamycin monotherapy in patients with type 1 diabetes modifies CD4(+)CD25(+)FOXP3(+) regulatory T-Cells. DIABETES, 57(9), 2341–2347.
- Activation of the aryl hydrocarbon receptor promotes allograft-specific tolerance through direct and dendritic cell-mediated effects on regulatory T cells
  Hauben, E., Gregori, S., Draghici, E., Migliavacca, B., Olivieri, S., Woisetschlaeger, M., & Roncarolo, M. G. (2008). Activation of the aryl hydrocarbon receptor promotes allograft-specific tolerance through direct and dendritic cell-mediated effects on regulatory T cells. BLOOD, 112(4), 1214–1222.
- Construction of human-SCID chimeric mice.
  Roncarolo, M. G., & Carballido, J. M. (2001). Construction of human-SCID chimeric mice. Current Protocols in Immunology / Edited by John E. Coligan ... [Et Al.], Chapter 4, Unit 4 8-?
- Is FOXP3 a bona fide marker for human regulatory T cells?
  Roncarolo, M.-G., & Gregori, S. (2008). Is FOXP3 a bona fide marker for human regulatory T cells? EUROPEAN JOURNAL OF IMMUNOLOGY, 38(4), 925–927.
- [Biomedical research: a need or a luxury?].
  Roncarolo, M. G. (2008). [Biomedical research: a need or a luxury?]. KOS, 25(264), 53–61.
- STAT5-signaling cytokines regulate the expression of FOXP3 in CD4(+)CD25(+) regulatory T cells and CD4(+)CD25(-) effector T cells
  Passerini, L., Allan, S. E., Battaglia, M., Di Nunzio, S., Alstad, A. N., Levings, M. K., … Bacchetta, R. (2008). STAT5-signaling cytokines regulate the expression of FOXP3 in CD4(+)CD25(+) regulatory T cells and CD4(+)CD25(-) effector T cells. INTERNATIONAL IMMUNOLOGY, 20(3), 421–431.
- Altered intracellular and extracellular signaling leads to impaired T-cell functions in ADA-SCID patients
  Cassani, B., Mirolo, M., Cattaneo, F., Benninghoff, U., Hershfield, M., Carlucci, F., … Aiuti, A. (2008). Altered intracellular and extracellular signaling leads to impaired T-cell functions in ADA-SCID patients. BLOOD, 111(8), 4209–4219.
- Development of lentiviral gene therapy for Wiskott Aldrich syndrome
  Galy, A., Roncarolo, M.-G., & Thrasher, A. J. (2008). Development of lentiviral gene therapy for Wiskott Aldrich syndrome. EXPERT OPINION ON BIOLOGICAL THERAPY, 8(2), 181–190.
- The role of tissue adaptation and graft size in immune tolerance
  Hauben, E., Roncarolo, M. G., Draghici, E., & Nevo, U. (2007). The role of tissue adaptation and graft size in immune tolerance. TRANSPLANT IMMUNOLOGY, 18(2), 122–125.
- Progress and prospects: Gene therapy clinical trials (part 2)
  Alton, E., Ferrari, S., & Griesenbach, U. (2007). Progress and prospects: Gene therapy clinical trials (part 2). GENE THERAPY, 14(22), 1555–1563.
- Generation of potent and stable human CD4(+) T regulatory cells by activation-independent expression of FOXP3
  Allan, S. E., Alstad, A. N., Merindol, N., Crellin, N. K., Amendola, M., Bacchetta, R., … Levings, M. K. (2008). Generation of potent and stable human CD4(+) T regulatory cells by activation-independent expression of FOXP3. MOLECULAR THERAPY, 16(1), 194–202.
- Immunological lessons learnt from patients transplanted with fully mismatched stem cells
  Touraine, J.-L., Plotnicky, H., Roncarolo, M.-G., Bacchetta, R., & Gebuhrer, L. (2007). Immunological lessons learnt from patients transplanted with fully mismatched stem cells. IMMUNOLOGIC RESEARCH, 38(1-3), 201–209.
- Isolation, expansion, and characterization of human natural and adaptive regulatory T cells.
  Gregori, S., Bacchetta, R., Passerini, L., Levings, M. K., & Roncarolo, M. G. (2007). Isolation, expansion, and characterization of human natural and adaptive regulatory T cells. Methods in Molecular Biology (Clifton, N.J.), 380, 83–105.
- Frequency and targeted detection of HLA-DPB1 T cell epitope disparities relevant in unrelated hematopoietic stem cell transplantation
  Zino, E., Vago, L., Di Terlizzi, S., Mazzi, B., Zito, L., Sironi, E., … Fleischhauer, K. (2007). Frequency and targeted detection of HLA-DPB1 T cell epitope disparities relevant in unrelated hematopoietic stem cell transplantation. BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 13(9), 1031–1040.
- Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy
  Aiuti, A., Cassani, B., Andolfi, G., Mirolo, M., Biasco, L., Recchia, A., … Bordignon, C. (2007). Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy. JOURNAL OF CLINICAL INVESTIGATION, 117(8), 2233–2240.
- Role of regulatory T cells and FOXP3 in human diseases
  Bacchetta, R., Gambineri, E., & Roncarolo, M.-G. (2007). Role of regulatory T cells and FOXP3 in human diseases. JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY, 120(2), 227–235.
- Regulatory T-cell immunotherapy for tolerance to self antigens and alloantigens in humans
  Roncarolo, M.-G., & Battaglia, M. (2007). Regulatory T-cell immunotherapy for tolerance to self antigens and alloantigens in humans. NATURE REVIEWS IMMUNOLOGY, 7(8), 585–598.
- The immune response to lentiviral-delivered transgene is modulated in vivo by transgene-expressing antigen-presenting cells but not by CD4(+)CD25(+) regulatory T cells
  Annoni, A., Battaglia, M., Follenzi, A., Lombardo, A., Sergi-Sergi, L., Naldini, L., & Roncarolo, M.-G. (2007). The immune response to lentiviral-delivered transgene is modulated in vivo by transgene-expressing antigen-presenting cells but not by CD4(+)CD25(+) regulatory T cells. BLOOD, 110(6), 1788–1796.
- A hypomorphic R229Q Rag2 mouse mutant recapitulates human Omenn syndrome
  Marrella, V., Poliani, P. L., Casati, A., Rucci, F., Frascoli, L., Gougeon, M.-L., … Villa, A. (2007). A hypomorphic R229Q Rag2 mouse mutant recapitulates human Omenn syndrome. JOURNAL OF CLINICAL INVESTIGATION, 117(5), 1260–1269.
- Activation-induced FOXP3 in human T effector cells does not suppress proliferation or cytokine production
  Allan, S. E., Crome, S. Q., Crellin, N. K., Passerini, L., Steiner, T. S., Bacchetta, R., … Levings, M. K. (2007). Activation-induced FOXP3 in human T effector cells does not suppress proliferation or cytokine production. INTERNATIONAL IMMUNOLOGY, 19(4), 345–354.
- Current understanding of the Wiskott-Aldrich syndrome and prospects for gene therapy.
  Trifari, S., Marangoni, F., Scaramuzza, S., Aiuti, A., Roncarolo, M. G., & Dupré, L. (2007). Current understanding of the Wiskott-Aldrich syndrome and prospects for gene therapy. Expert Review of Clinical Immunology, 3(2), 205–215.
- In vivo administration of lentiviral vectors triggers a type I interferon response that restricts hepatocyte gene transfer and promotes vector clearance
  Brown, B. D., Sitia, G., Annoni, A., Hauben, E., Sergi, L. S., Zingale, A., … Naldini, L. (2007). In vivo administration of lentiviral vectors triggers a type I interferon response that restricts hepatocyte gene transfer and promotes vector clearance. BLOOD, 109(7), 2797–2805.
- Rapamycin promotes expansion of functional CD4(+)CD25(+)FOXP3(+) regulatory T cells of both healthy subjects and type 1 diabetic patients
  Battaglia, M., Stabilini, A., Migliavacca, B., Horejs-Hoeck, J., Kaupper, T., & Roncarolo, M.-G. (2006). Rapamycin promotes expansion of functional CD4(+)CD25(+)FOXP3(+) regulatory T cells of both healthy subjects and type 1 diabetic patients. JOURNAL OF IMMUNOLOGY, 177(12), 8338–8347.
- Defective Th1 cytokine gene transcription in CD4(+) and CD8(+) T cells from Wiskott-Aldrich syndrome patients
  Trifari, S., Sitia, G., Aiuti, A., Scaramuzza, S., Marangoni, F., Guidotti, L. G., … Dupre, L. (2006). Defective Th1 cytokine gene transcription in CD4(+) and CD8(+) T cells from Wiskott-Aldrich syndrome patients. JOURNAL OF IMMUNOLOGY, 177(10), 7451–7461.
- Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients
  Charrier, S., Dupre, L., Scaramuzza, S., Jeanson-Leh, L., Blundell, M. P., Danos, O., … Galy, A. (2007). Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients. GENE THERAPY, 14(5), 415–428.
- Induction of transplantation tolerance via regulatory T cells.
  Battaglia, M., & Roncarolo, M. G. (2006). Induction of transplantation tolerance via regulatory T cells. Inflammation & Allergy Drug Targets, 5(3), 157–165.
- Interleukin-10-secreting type 1 regulatory T cells in rodents and humans
  Roncarolo, M. G., Gregori, S., Battaglia, M., Bacchetta, R., Fleischhauer, K., & Levings, M. K. (2006). Interleukin-10-secreting type 1 regulatory T cells in rodents and humans. IMMUNOLOGICAL REVIEWS, 212, 28–50.
- Ex vivo gene therapy with lentiviral vectors rescues adenosine deaminase (ADA)-deficient mice and corrects their immune and metabolic defects
  Mortellaro, A., Hernandez, R. J., Guerrini, M. M., Carlucci, F., Tabucchi, A., Ponzoni, M., … Aiuti, A. (2006). Ex vivo gene therapy with lentiviral vectors rescues adenosine deaminase (ADA)-deficient mice and corrects their immune and metabolic defects. BLOOD, 108(9), 2979–2988.
- Defective regulatory and effector T cell functions in patients with FOXP3 mutations
  Bacchetta, R., Passerini, L., Gambineri, E., Dai, M., Allan, S. E., Perroni, L., … Roncarolo, M. G. (2006). Defective regulatory and effector T cell functions in patients with FOXP3 mutations. JOURNAL OF CLINICAL INVESTIGATION, 116(6), 1713–1722.
- Design and optimization of lentiviral vectors for transfer of GALC expression in Twitcher brain
  Dolcetta, D., Perani, L., Givogri, M. I., Galbiati, F., Amadio, S., Del Carro, U., … Bongarzone, E. (2006). Design and optimization of lentiviral vectors for transfer of GALC expression in Twitcher brain. JOURNAL OF GENE MEDICINE, 8(8), 962–971.
- Induction of tolerance in type 1 diabetes via both CD4(+) CD25(+) T regulatory cells and T regulatory type 1 cells
  Battaglia, M., Stabilini, A., Draghici, E., Migliavacca, B., Gregori, S., Bonifacio, E., & Roncarolo, M.-G. (2006). Induction of tolerance in type 1 diabetes via both CD4(+) CD25(+) T regulatory cells and T regulatory type 1 cells. DIABETES, 55(6), 1571–1580.
- Efficacy of gene therapy for Wiskott-Aldrich syndrome using a WAS promoter/cDNA-containing lentiviral vector and nonlethal irradiation
  Dupre, L., Marangoni, F., Scaramuzza, S., Trifari, S., Hernandez, R. J., Aiuti, A., … Roncarolo, M. G. (2006). Efficacy of gene therapy for Wiskott-Aldrich syndrome using a WAS promoter/cDNA-containing lentiviral vector and nonlethal irradiation. HUMAN GENE THERAPY, 17(3), 303–313.
- Rapamycin and interleukin-10 treatment induces T regulatory type 1 cells that mediate antigen-specific transplantation tolerance
  Battaglia, M., Stabilini, A., Draghici, E., Gregori, S., Mocchetti, C., Bonifacio, E., & Roncarolo, M. G. (2006). Rapamycin and interleukin-10 treatment induces T regulatory type 1 cells that mediate antigen-specific transplantation tolerance. DIABETES, 55(1), 40–49.
- Regulatory T cells: prospective for clinical application in hematopoietic stem cell transplantation
  Gregori, S., Bacchetta, R., Hauben, E., Battaglia, M., & Roncarolo, M. G. (2005). Regulatory T cells: prospective for clinical application in hematopoietic stem cell transplantation. CURRENT OPINION IN HEMATOLOGY, 12(6), 451–456.
- CD4(+) regulatory T cells: Mechanisms of induction and effector function
  Bacchetta, R., Gregori, S., & Roncarolo, M. G. (2005). CD4(+) regulatory T cells: Mechanisms of induction and effector function. AUTOIMMUNITY REVIEWS, 4(8), 491–496.
- The role of 2 FOXP3 isoforms in the generation of human CD4(+) Tregs
  Allan, S. E., Passerini, L., Bacchetta, R., Crellin, N., Dai, M. Y., ORBAN, P. C., … Levings, M. K. (2005). The role of 2 FOXP3 isoforms in the generation of human CD4(+) Tregs. JOURNAL OF CLINICAL INVESTIGATION, 115(11), 3276–3284.
- Utilizing regulatory T cells to control alloreactivity
  Hauben, E., Bacchetta, R., & Roncarolo, M. G. (2005). Utilizing regulatory T cells to control alloreactivity. CYTOTHERAPY, 7(2), 158–165.
- Phenotypic and functional differences between human CD4(+)CD25(+) and type 1 regulatory T cells
  Levings, A. K., & Roncarolo, M. G. (2005). Phenotypic and functional differences between human CD4(+)CD25(+) and type 1 regulatory T cells. CD4-PLUSCD25-PLUS REGULATORY T CELLS: ORIGIN, FUNCTION AND THERAPEUTIC POTENTIAL, 293, 303–326.
- Myelin deterioration in twitcher mice: Motor evoked potentials and magnetic resonance imaging as in vivo monitoring tools
  Dolcetta, D., Amadio, S., Guerrini, U., Givogri, M. I., Perani, L., Galbiati, F., … Bongarzone, E. (2005). Myelin deterioration in twitcher mice: Motor evoked potentials and magnetic resonance imaging as in vivo monitoring tools. JOURNAL OF NEUROSCIENCE RESEARCH, 81(4), 597–604.
- Human CD4+regulatory T cells and activation-induced tolerance
  Hauben, E., & Roncarolo, M. G. (2005). Human CD4+regulatory T cells and activation-induced tolerance. MICROBES AND INFECTION, 7(7-8), 1023–1032.
- An anti-CD45RO/RB monoclonal antibody modulates T cell responses via induction of apoptosis and generation of regulatory T cells
  Gregori, S., Mangia, P., Bacchetta, R., Tresoldi, E., Kolbinger, F., Traversari, C., … Roncarolo, M. G. (2005). An anti-CD45RO/RB monoclonal antibody modulates T cell responses via induction of apoptosis and generation of regulatory T cells. JOURNAL OF EXPERIMENTAL MEDICINE, 201(8), 1293–1305.
- Induction of transplantation tolerance in humans using fetal cell transplants
  Touraine, J. L., Roncarolo, M. G., Raudrant, D., Bacchetta, R., Golfier, F., Sembeil, R., & Gebuhrer, L. (2005). Induction of transplantation tolerance in humans using fetal cell transplants. TRANSPLANTATION PROCEEDINGS, 37(1), 65–66.
- Impaired humoral immunity in X-linked lymphoproliferative disease is associated with defective IL-10 production by CD4+ T cells
  Ma, C. S., Hare, N. J., Nichols, K. E., Dupre, L., Andolfi, G., Roncarolo, M. G., … Tangye, S. G. (2005). Impaired humoral immunity in X-linked lymphoproliferative disease is associated with defective IL-10 production by CD4+ T cells. JOURNAL OF CLINICAL INVESTIGATION, 115(4), 1049–1059.
- Rapamycin selectively expands CD4(+)CD25(+)FoxP3(+) regulatory T cells
  Battaglia, M., Stabilini, A., & Roncarolo, M. G. (2005). Rapamycin selectively expands CD4(+)CD25(+)FoxP3(+) regulatory T cells. BLOOD, 105(12), 4743–4748.
- A proportion of patients with lymphoma may harbor mutations of the perforin gene
  Clementi, R., Locatelli, F., Dupre, L., Garaventa, A., Emmi, L., Bregni, M., … Roncarolo, M. G. (2005). A proportion of patients with lymphoma may harbor mutations of the perforin gene. BLOOD, 105(11), 4424–4428.
- IL-10-producing T regulatory type 1 cells and oral tolerance
  Battaglia, M., Gianfrani, C., Gregori, S., & Roncarolo, M. G. (2004). IL-10-producing T regulatory type 1 cells and oral tolerance. Presented at the Conference on Oral Tolerance, NEW YORK,NY: NEW YORK ACAD SCIENCES.
- SAP controls the cytolytic activity of CD8(+) T cells against EBV-infected cells
  Dupre, L., Andolfi, G., Tangye, S. G., Clementi, R., Locatelli, F., Arico, M., … Roncarolo, M. G. (2005). SAP controls the cytolytic activity of CD8(+) T cells against EBV-infected cells. BLOOD, 105(11), 4383–4389.
- Recombinant human interleukin 10 suppresses gliadin dependent T cell activation in ex vivo cultured coeliac intestinal mucosa
  Salvati, V. M., Mazzarella, G., Gianfrani, C., Levings, M. K., Stefanile, R., De Giulio, B., … Troncone, R. (2005). Recombinant human interleukin 10 suppresses gliadin dependent T cell activation in ex vivo cultured coeliac intestinal mucosa. GUT, 54(1), 46–53.
- IL-3 or IL-7 increases ex vivo gene transfer efficiency in ADA-SCID BM CD34(+) cells while maintaining in vivo lymphoid potential
  Ficara, F., Superchi, D. B., Hernandez, R. J., Mocchetti, C., Carballido-Perrig, N., Andolfi, G., … Aiuti, A. (2004). IL-3 or IL-7 increases ex vivo gene transfer efficiency in ADA-SCID BM CD34(+) cells while maintaining in vivo lymphoid potential. MOLECULAR THERAPY, 10(6), 1096–1108.
- Lentiviral vector-mediated gene transfer in T cells from Wiskott-Aldrich syndrome patients leads to functional correction
  Dupre, L., Trifari, S., Follenzi, A., Marangoni, F., de Lera, T. L., Bernad, A., … Roncarolo, M. G. (2004). Lentiviral vector-mediated gene transfer in T cells from Wiskott-Aldrich syndrome patients leads to functional correction. MOLECULAR THERAPY, 10(5), 903–915.
- Differentiation of Tr1 cells by immature dendritic cells requires IL-10 but not CD25(+)CD4(+) Tr cells
  Levings, M. K., Gregori, S., Tresoldi, E., Cazzaniga, S., Bonini, C., & Roncarolo, M. G. (2005). Differentiation of Tr1 cells by immature dendritic cells requires IL-10 but not CD25(+)CD4(+) Tr cells. BLOOD, 105(3), 1162–1169.
- Analysis of galactocerebrosidase activity in the mouse brain by a new histological staining method
  Dolcetta, D., Perani, L., Givogri, M. I., Galbiati, F., Orlacchio, A., Martino, S., … Bongarzone, E. (2004). Analysis of galactocerebrosidase activity in the mouse brain by a new histological staining method. JOURNAL OF NEUROSCIENCE RESEARCH, 77(3), 462–464.
- Reappraisal of in utero stem cell transplantation based on long-term results
  Touraine, J. L., Raudrant, D., Golfier, F., Rebaud, A., Sembeil, R., Roncarolo, M. G., … Gebuhrer, L. (2004). Reappraisal of in utero stem cell transplantation based on long-term results. FETAL DIAGNOSIS AND THERAPY, 19(4), 305–312.
- Efficient gene transfer into primitive hematopoietic progenitors using a bone marrow microenvironment cell line engineered to produce retroviral vectors
  Dando, J. S., Ficara, F., Deola, S., Roncarolo, M. G., Bordignon, C., & Aiuti, A. (2004). Efficient gene transfer into primitive hematopoietic progenitors using a bone marrow microenvironment cell line engineered to produce retroviral vectors. HAEMATOLOGICA, 89(4), 462–470.
- Mobilized blood CD341 cells transduced and selected with a clinically applicable protocol reconstitute lymphopoiesis in SCID-Hu mice
  Deola, S., Scaramuzza, S., Birolo, R. S., Carballido-Perrig, N., Ficara, F., Mocchetti, C., … Aiuti, A. (2004). Mobilized blood CD341 cells transduced and selected with a clinically applicable protocol reconstitute lymphopoiesis in SCID-Hu mice. HUMAN GENE THERAPY, 15(3), 305–311.
- The role of cytokines (and not only) in inducing and expanding T regulatory type 1 cells
  Battaglia, M., & Roncarolo, M. G. (2004). The role of cytokines (and not only) in inducing and expanding T regulatory type 1 cells. TRANSPLANTATION, 77(1), S16–S18.
- Targeting lentiviral vector expression to hepatocytes limits transgene-specific immune response and establishes long-term expression of human antihemophilic factor IX in mice
  Follenzi, A., Battaglia, M., Lombardo, A., Annoni, A., Roncarolo, M. G., & Naldini, L. (2004). Targeting lentiviral vector expression to hepatocytes limits transgene-specific immune response and establishes long-term expression of human antihemophilic factor IX in mice. BLOOD, 103(10), 3700–3709.
- Gene therapy for adenosine deaminase deficiency.
  Aiuti, A., Ficara, F., Cattaneo, F., Bordignon, C., & Roncarolo, M. G. (2003). Gene therapy for adenosine deaminase deficiency. Current Opinion in Allergy and Clinical Immunology, 3(6), 461–466.
- Type 1 T regulatory cells and their relationship with CD4+CD25+ T regulatory cells.
  Roncarolo, M. G., Gregori, S., & Levings, M. (2003). Type 1 T regulatory cells and their relationship with CD4+CD25+ T regulatory cells. Novartis Foundation Symposium, 252, 115–127.
- The role of interleukin 10 in the control of autoimmunity
  Roncarolo, M. G., Battaglia, M., & Gregori, S. (2003). The role of interleukin 10 in the control of autoimmunity. JOURNAL OF AUTOIMMUNITY, 20(4), 269–272.
- IL-10 and TGF-beta induce alloreactive CD4(+)CD25(-) T cells to acquire regulatory cell function
  Chen, Z. M., O'Shaughnessy, M. J., Gramaglia, I., Panoskaltsis-Mortari, A., Murphy, W. J., Narula, S., … Blazar, B. R. (2003). IL-10 and TGF-beta induce alloreactive CD4(+)CD25(-) T cells to acquire regulatory cell function. BLOOD, 101(12), 5076–5083.
- The role of IL-10 and TGF-beta in the differentiation and effector function of T regulatory cells
  Levings, M. K., Bacchetta, R., Schulz, U., & Roncarolo, M. G. (2002). The role of IL-10 and TGF-beta in the differentiation and effector function of T regulatory cells. INTERNATIONAL ARCHIVES OF ALLERGY AND IMMUNOLOGY, 129(4), 263–276.
- Human CD25(+) CD4(+) T suppressor cell clones produce transforming growth factor beta, but not interleukin 10, and are distinct from type 1 T regulatory cells
  Levings, M. K., Sangregorio, R., Sartirana, C., Moschin, A. L., Battaglia, M., ORBAN, P. C., & Roncarolo, M. G. (2002). Human CD25(+) CD4(+) T suppressor cell clones produce transforming growth factor beta, but not interleukin 10, and are distinct from type 1 T regulatory cells. JOURNAL OF EXPERIMENTAL MEDICINE, 196(10), 1335–1346.
- Growth and expansion of human T regulatory type 1 cells are independent from TCR activation but require exogenous cytokines
  Bacchetta, R., Sartirana, C., Levings, M. K., Bordignon, C., Narula, S., & Roncarolo, M. G. (2002). Growth and expansion of human T regulatory type 1 cells are independent from TCR activation but require exogenous cytokines. EUROPEAN JOURNAL OF IMMUNOLOGY, 32(8), 2237–2245.
- Wiskott-Aldrich syndrome protein regulates lipid raft dynamics during immunological synapse formation
  Dupre, L., Aiuti, A., Trifari, S., Martino, S., Saracco, P., Bordignon, C., & Roncarolo, M. G. (2002). Wiskott-Aldrich syndrome protein regulates lipid raft dynamics during immunological synapse formation. IMMUNITY, 17(2), 157–166.
- Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement
  Aiuti, A., Vai, S., Mortellaro, A., Casorati, G., Ficara, F., Andolfi, G., … Bordignon, C. (2002). Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement. NATURE MEDICINE, 8(5), 423–425.
- The puzzling world of murine T regulatory cells
  Battaglia, M., Blazar, B. R., & Roncarolo, M. G. (2002). The puzzling world of murine T regulatory cells. MICROBES AND INFECTION, 4(5), 559–566.
- Therapeutic applications for hematopoietic stem cell gene transfer
  Bordignon, C., & Roncarolo, M. G. (2002). Therapeutic applications for hematopoietic stem cell gene transfer. NATURE IMMUNOLOGY, 3(4), 318–321.
- Type 1 T regulatory cells
  Roncarolo, M. G., Bacchetta, R., Bordignon, C., Narula, S., & Levings, M. K. (2001). Type 1 T regulatory cells. IMMUNOLOGICAL REVIEWS, 182, 68–79.
- A novel human packaging cell line with hematopoietic supportive capacity increases gene transfer into early hematopoietic progenitors
  Dando, J. S., Roncarolo, M. G., Bordignon, C., & Aiuti, A. (2001). A novel human packaging cell line with hematopoietic supportive capacity increases gene transfer into early hematopoietic progenitors. HUMAN GENE THERAPY, 12(16), 1979–1988.
- Human CD25(+)CD4(+) T regulatory cells suppress naive and memory T cell proliferation and can be expanded in vitro without loss of function
  Levings, M. K., Sangregorio, R., & Roncarolo, M. G. (2001). Human CD25(+)CD4(+) T regulatory cells suppress naive and memory T cell proliferation and can be expanded in vitro without loss of function. JOURNAL OF EXPERIMENTAL MEDICINE, 193(11), 1295–1301.
- IFN-alpha and IL-10 induce the differentiation of human type 1 T regulatory cells
  Levings, M. K., Sangregorio, R., Galbiati, F., Squadrone, S., Malefyt, R. D., & Roncarolo, M. G. (2001). IFN-alpha and IL-10 induce the differentiation of human type 1 T regulatory cells. JOURNAL OF IMMUNOLOGY, 166(9), 5530–5539.
- Differentiation of T regulatory cells by immature dendritic cells
  Roncarolo, M. G., Levings, M. K., & Traversari, C. (2001). Differentiation of T regulatory cells by immature dendritic cells. JOURNAL OF EXPERIMENTAL MEDICINE, 193(2), F5–F9.
- Altered T-cell receptor+CD28-mediated signaling and blocked cell cycle progression in interleukin 10 and transforming growth factor-beta-treated alloreactive T cells that do not induce graft-versus-host disease
  Boussiotis, V. A., Chen, Z. M., Zeller, J. C., Murphy, W. J., Berezovskaya, A., Narula, S., … Blazar, B. R. (2001). Altered T-cell receptor+CD28-mediated signaling and blocked cell cycle progression in interleukin 10 and transforming growth factor-beta-treated alloreactive T cells that do not induce graft-versus-host disease. BLOOD, 97(2), 565–571.
- The role of different subsets of T regulatory cells in controlling autoimmunity
  Roncarolo, M. G., & Levings, M. K. (2000). The role of different subsets of T regulatory cells in controlling autoimmunity. CURRENT OPINION IN IMMUNOLOGY, 12(6), 676–683.
- T cell receptor-dependent activation of human lymphocytes through cell surface ganglioside GT1b: implications for innate immunity
  Bukowski, J. F., Roncarolo, M. G., Spits, H., Krangel, M. S., Morita, C. T., Brenner, M. B., & Band, H. (2000). T cell receptor-dependent activation of human lymphocytes through cell surface ganglioside GT1b: implications for innate immunity. EUROPEAN JOURNAL OF IMMUNOLOGY, 30(11), 3199–3206.
- T-regulatory 1 cells: A novel subset of CD4(+)T cells with immunoregulatory properties
  Levings, M. K., & Roncarolo, M. G. (2000). T-regulatory 1 cells: A novel subset of CD4(+)T cells with immunoregulatory properties. JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY, 106(1), S109–S112.
- Prognostic significance of increased IL-10 production in patients prior to allogeneic bone marrow transplantation
  Holler, E., Roncarolo, M. G., Hintermeier-Knabe, R., Eissner, G., Ertl, B., Schulz, U., … Wilmanns, W. (2000). Prognostic significance of increased IL-10 production in patients prior to allogeneic bone marrow transplantation. BONE MARROW TRANSPLANTATION, 25(3), 237–241.
- Generation of primary antigen-specific human T- and B-cell responses in immunocompetent SCID-hu mice
  Carballido, J. M., Namikawa, R., Carballido-Perrig, N., Antonenko, S., Roncarolo, M. G., & de Vries, J. E. (2000). Generation of primary antigen-specific human T- and B-cell responses in immunocompetent SCID-hu mice. NATURE MEDICINE, 6(1), 103–106.
- IL-10 transgenic mice present a defect in T cell development reminiscent to SCID patients
  Rouleau, M., Cottrez, F., Bigler, M., Antonenko, S., Carballido, J. M., Zlotnik, A., … Groux, H. (1999). IL-10 transgenic mice present a defect in T cell development reminiscent to SCID patients. JOURNAL OF IMMUNOLOGY, 163(3), 1420–1427.
- High spontaneous IL-10 production in unrelated bone marrow transplant recipients is associated with fewer transplant-related complications and early deaths
  Baker, K. S., Roncarolo, M. G., Peters, C., Bigler, M., DeFor, T., & Blazar, B. R. (1999). High spontaneous IL-10 production in unrelated bone marrow transplant recipients is associated with fewer transplant-related complications and early deaths. BONE MARROW TRANSPLANTATION, 23(11), 1123–1129.
- Administration of Flk2/Flt3 ligand induces expansion of human high-proliferative potential colony-forming cells in the SCID-hu mouse
  Namikawa, R., Muench, M. O., Firpo, M. T., Humeau, L., Xu, Y. M., Menon, S., & Roncarolo, M. G. (1999). Administration of Flk2/Flt3 ligand induces expansion of human high-proliferative potential colony-forming cells in the SCID-hu mouse. EXPERIMENTAL HEMATOLOGY, 27(6), 1029–1037.
- Ex vivo manipulations alter the reconstitution potential of mobilized human CD34(+) peripheral blood progenitors
  Humeau, L., Namikawa, R., Bardin, F., Mannoni, P., Roncarolo, M. G., & Chabannon, C. (1999). Ex vivo manipulations alter the reconstitution potential of mobilized human CD34(+) peripheral blood progenitors. LEUKEMIA, 13(3), 438–452.
- A transgenic model to analyze the immunoregulatory role of IL-10 secreted by antigen-presenting cells
  Groux, H., Cottrez, F., Rouleau, M., Mauze, S., Antonenko, S., Hurst, S., … Coffman, R. L. (1999). A transgenic model to analyze the immunoregulatory role of IL-10 secreted by antigen-presenting cells. JOURNAL OF IMMUNOLOGY, 162(3), 1723–1729.
- Interleukin-10 dose-dependent regulation of CD4(+) and CD8(+) T cell-mediated graft-versus-host disease
  Blazar, B. R., Taylor, P. A., Panoskaltsis-Mortari, A., Narula, S. K., Smith, S. R., Roncarolo, M. G., & Vallera, D. A. (1998). Interleukin-10 dose-dependent regulation of CD4(+) and CD8(+) T cell-mediated graft-versus-host disease. TRANSPLANTATION, 66(9), 1220–1229.
- The X-linked lymphoproliferative-disease gene product SAP regulates signals induced through the co-receptor SLAM
  Sayos, J., Wu, C., Morra, M., Wang, N., Zhang, X., Allen, D., … Terhorst, C. (1998). The X-linked lymphoproliferative-disease gene product SAP regulates signals induced through the co-receptor SLAM. NATURE, 395(6701), 462–469.
- Transgene expression of bcl-x(L) permits anti-immunoglobulin (Ig)-induced proliferation in xid B cells
  Solvason, N., Wu, W. W., Kabra, N., Lund-Johansen, F., Roncarolo, M. G., Behrens, T. W., … Howard, M. (1998). Transgene expression of bcl-x(L) permits anti-immunoglobulin (Ig)-induced proliferation in xid B cells. JOURNAL OF EXPERIMENTAL MEDICINE, 187(7), 1081–1091.
- Inhibitory and stimulatory effects of IL-10 on human CD8(+) T cells
  Groux, H., Bigler, M., de Vries, J. E., & Roncarolo, M. G. (1998). Inhibitory and stimulatory effects of IL-10 on human CD8(+) T cells. JOURNAL OF IMMUNOLOGY, 160(7), 3188–3193.
- Successful reconstitution of human hematopoiesis in the SCID-hu mouse by genetically modified, highly enriched progenitors isolated from fetal liver
  Humeau, L., Chabannon, C., Firpo, M. T., Mannoni, P., Bagnis, C., Roncarolo, M. G., & Namikawa, R. (1997). Successful reconstitution of human hematopoiesis in the SCID-hu mouse by genetically modified, highly enriched progenitors isolated from fetal liver. BLOOD, 90(9), 3496–3506.
- A CD4(+) T-cell subset inhibits antigen-specific T-cell responses and prevents colitis
  Groux, H., OGARRA, A., Bigler, M., Rouleau, M., Antonenko, S., deVries, J. E., & Roncarolo, M. G. (1997). A CD4(+) T-cell subset inhibits antigen-specific T-cell responses and prevents colitis. NATURE, 389(6652), 737–742.
- Immunological tolerance following stem cell transplantation in human fetuses in utero
  TOURAINE, J. L., Raudrant, D., Laplace, S., & Roncarolo, M. G. (1997). Immunological tolerance following stem cell transplantation in human fetuses in utero. TRANSPLANTATION PROCEEDINGS, 29(5), 2477–2477.
- Induction of human T helper cell type 1 differentiation results in loss of IFN-gamma receptor beta-chain expression
  Groux, H., Sornasse, T., Cottrez, F., deVries, J. E., Coffman, R. L., Roncarolo, M. G., & Yssel, H. (1997). Induction of human T helper cell type 1 differentiation results in loss of IFN-gamma receptor beta-chain expression. JOURNAL OF IMMUNOLOGY, 158(12), 5627–5631.
- Inflammatory reactions induced by pretransplant conditioning - An alternative target for modulation of acute GVHD and complications following allogeneic bone marrow transplantation?
  Holler, E., Ertl, B., HINTERMEIERKNABE, R., Roncarolo, M. G., Eissner, G., Mayer, F., … Wilmanns, W. (1997). Inflammatory reactions induced by pretransplant conditioning - An alternative target for modulation of acute GVHD and complications following allogeneic bone marrow transplantation? LEUKEMIA & LYMPHOMA, 25(3-4), 217–224.
- Colony-forming cells expressing high levels of CD34 are the main targets for granulocyte colony-stimulating factor and macrophage colony-stimulating factor in the human fetal liver
  Muench, M. O., Roncarolo, M. G., ROSNET, O., Birnbaum, D., & Namikawa, R. (1997). Colony-forming cells expressing high levels of CD34 are the main targets for granulocyte colony-stimulating factor and macrophage colony-stimulating factor in the human fetal liver. EXPERIMENTAL HEMATOLOGY, 25(4), 277–287.
- Phenotypic and functional evidence for the expression of CD4 by hematopoietic stem cells isolated from human fetal liver
  Muench, M. O., Roncarolo, M. G., & Namikawa, R. (1997). Phenotypic and functional evidence for the expression of CD4 by hematopoietic stem cells isolated from human fetal liver. BLOOD, 89(4), 1364–1375.
- Antigen-specific cytotoxic T cells mediate human fetal pancreas allograft rejection in SCID-hu mice
  Rouleau, M., Namikawa, R., Antonenko, S., CARBALLIDOPERRIG, N., & Roncarolo, M. G. (1996). Antigen-specific cytotoxic T cells mediate human fetal pancreas allograft rejection in SCID-hu mice. JOURNAL OF IMMUNOLOGY, 157(12), 5710–5720.
- Treatment of X-linked severe combined immunodeficiency by in utero transplantation of paternal bone marrow
  Flake, A. W., Roncarolo, M. G., Puck, J. M., ALMEIDAPORADA, G., Evans, M. I., Johnson, M. P., … Zanjani, E. D. (1996). Treatment of X-linked severe combined immunodeficiency by in utero transplantation of paternal bone marrow. NEW ENGLAND JOURNAL OF MEDICINE, 335(24), 1806–1810.
- Human T-and B-cell functions in SCID-hu mice.
  Roncarolo, M. G., Carballido, J. M., Rouleau, M., Namikawa, R., & de Vries, J. E. (1996). Human T-and B-cell functions in SCID-hu mice. Seminars in Immunology, 8(4), 207–213.
- Regulatory roles of the ligand for Flk2/Flt3 tyrosine kinase receptor on human hematopoiesis
  Namikawa, R., Muench, M. O., & Roncarolo, M. G. (1996). Regulatory roles of the ligand for Flk2/Flt3 tyrosine kinase receptor on human hematopoiesis. STEM CELLS, 14(4), 388–395.
- Interleukin-10 induces a long-term antigen-specific anergic state in human CD4(+) T cells
  Groux, H., Bigler, M., deVries, J. E., & Roncarolo, M. G. (1996). Interleukin-10 induces a long-term antigen-specific anergic state in human CD4(+) T cells. JOURNAL OF EXPERIMENTAL MEDICINE, 184(1), 19–29.
- Immune functions of cord blood cells before and after transplantation.
  Roncarolo, M. G., Bigler, M., Martino, S., CIUTI, E., Tovo, P. A., & Wagner, J. (1996). Immune functions of cord blood cells before and after transplantation. Journal of Hematotherapy, 5(2), 157–160.
- The FLK2/FLT3 ligand synergizes with interleukin-7 in promoting stromal-cell-independent expansion and differentiation of human fetal pro-B cells in vitro
  Namikawa, R., Muench, M. O., deVries, J. E., & Roncarolo, M. G. (1996). The FLK2/FLT3 ligand synergizes with interleukin-7 in promoting stromal-cell-independent expansion and differentiation of human fetal pro-B cells in vitro. BLOOD, 87(5), 1881–1890.
- T-cell subsets and their cytokine profiles in transplantation and tolerance
  Groux, H., Rouleau, M., Bacchetta, R., & Roncarolo, M. G. (1995). T-cell subsets and their cytokine profiles in transplantation and tolerance. Presented at the Conference on Bone Marrow Transplantation in the 90s - Into the 21st-Century, ORLANDO,FL: NEW YORK ACAD SCIENCES.
- IL-4 INDUCES HUMAN B-CELL MATURATION AND IGE SYNTHESIS IN SCID-HU MICE - INHIBITION OF ONGOING IGE PRODUCTION BY IN-VIVO TREATMENT WITH AN IL-4/IL-13 RECEPTOR ANTAGONIST
  Carballido, J. M., SCHOLS, D., Namikawa, R., Zurawski, S., Zurawski, G., Roncarolo, M. G., & deVries, J. E. (1995). IL-4 INDUCES HUMAN B-CELL MATURATION AND IGE SYNTHESIS IN SCID-HU MICE - INHIBITION OF ONGOING IGE PRODUCTION BY IN-VIVO TREATMENT WITH AN IL-4/IL-13 RECEPTOR ANTAGONIST. JOURNAL OF IMMUNOLOGY, 155(9), 4162–4170.
- HLA-HAPLOIDENTICAL UMBILICAL-CORD BLOOD STEM-CELL TRANSPLANTATION IN A CHILD WITH ADVANCED LEUKEMIA - CLINICAL OUTCOME AND ANALYSIS OF HEMATOPOIETIC RECOVERY
  Miniero, R., Busca, A., Roncarolo, M. G., Saitta, M., Iavarone, A., Timeus, F., … Madon, E. (1995). HLA-HAPLOIDENTICAL UMBILICAL-CORD BLOOD STEM-CELL TRANSPLANTATION IN A CHILD WITH ADVANCED LEUKEMIA - CLINICAL OUTCOME AND ANALYSIS OF HEMATOPOIETIC RECOVERY. BONE MARROW TRANSPLANTATION, 16(2), 229–240.
- DYSFUNCTIONAL CYTOKINE PRODUCTION BY HOST-REACTIVE T-CELL - CLONES ISOLATED FROM A CHIMERIC SEVERE COMBINED IMMUNODEFICIENCY PATIENT TRANSPLANTED WITH HAPLOIDENTICAL BONE-MARROW
  Bacchetta, R., Parkman, R., McMahon, M., Weinberg, K., Bigler, M., deVries, J. E., & Roncarolo, M. G. (1995). DYSFUNCTIONAL CYTOKINE PRODUCTION BY HOST-REACTIVE T-CELL - CLONES ISOLATED FROM A CHIMERIC SEVERE COMBINED IMMUNODEFICIENCY PATIENT TRANSPLANTED WITH HAPLOIDENTICAL BONE-MARROW. BLOOD, 85(7), 1944–1953.
- UNIQUE CYTOKINE PRODUCTION PROFILE OF ANERGIC HUMAN T-CELLS IN SCID-HU MICE AFTER STAPHYLOCOCCAL-ENTEROTOXIN-B ADMINISTRATION
  SCHOLS, D., Jones, D., & Roncarolo, M. G. (1995). UNIQUE CYTOKINE PRODUCTION PROFILE OF ANERGIC HUMAN T-CELLS IN SCID-HU MICE AFTER STAPHYLOCOCCAL-ENTEROTOXIN-B ADMINISTRATION. JOURNAL OF IMMUNOLOGY, 154(7), 3204–3212.
- TRACING THE EXPRESSION OF CD7 AND OTHER ANTIGENS DURING T-CELL AND MYELOID-CELL DIFFERENTIATION IN THE HUMAN FETAL LIVER AND THYMUS
  Barcena, A., Muench, M. O., Roncarolo, M. G., & Spits, H. (1995). TRACING THE EXPRESSION OF CD7 AND OTHER ANTIGENS DURING T-CELL AND MYELOID-CELL DIFFERENTIATION IN THE HUMAN FETAL LIVER AND THYMUS. LEUKEMIA & LYMPHOMA, 17(1-2), 1–11.
- FLK-2/FLT-3 LIGAND REGULATES THE GROWTH OF EARLY MYELOID PROGENITORS ISOLATED FROM HUMAN FETAL LIVER
  Muench, M. O., Roncarolo, M. G., Menon, S., Xu, Y. M., Kastelein, R., Zurawski, S., … Namikawa, R. (1995). FLK-2/FLT-3 LIGAND REGULATES THE GROWTH OF EARLY MYELOID PROGENITORS ISOLATED FROM HUMAN FETAL LIVER. BLOOD, 85(4), 963–972.
- TRANSPLANTATION OF MISMATCHED HUMAN FETAL LIVER-CELLS - TOLERANCE INDUCTION VIA CLONAL DELETION AND CLONAL ANERGY
  TOURAINE, J. L., Bacchetta, R., Yssel, H., Devries, J., & Roncarolo, M. G. (1995). TRANSPLANTATION OF MISMATCHED HUMAN FETAL LIVER-CELLS - TOLERANCE INDUCTION VIA CLONAL DELETION AND CLONAL ANERGY. TRANSPLANTATION PROCEEDINGS, 27(1), 622–624.
- PROGRESS IN THE EX-VIVO EXPANSION OF HEMATOPOIETIC PROGENITORS
  Muench, M. O., Roncarolo, M. G., Namikawa, R., Barcena, A., & Moore, M. A. S. (1994). PROGRESS IN THE EX-VIVO EXPANSION OF HEMATOPOIETIC PROGENITORS. LEUKEMIA & LYMPHOMA, 16(1-2), 1–11.
- ANTI-SCID MOUSE REACTIVITY SHAPES THE HUMAN CD4(+) T-CELL REPERTOIRE IN HU-P8L-SCID CHIMERAS
  TARYLEHMANN, M., Lehmann, P. V., SCHOLS, D., Roncarolo, M. G., & Saxon, A. (1994). ANTI-SCID MOUSE REACTIVITY SHAPES THE HUMAN CD4(+) T-CELL REPERTOIRE IN HU-P8L-SCID CHIMERAS. JOURNAL OF EXPERIMENTAL MEDICINE, 180(5), 1817–1827.
- IDENTIFICATION OF A COMMON T-NATURAL-KILLER-CELL PROGENITOR IN HUMAN FETAL THYMUS
  Sanchez, M. J., Muench, M. O., Roncarolo, M. G., Lanier, L. L., & Phillips, J. H. (1994). IDENTIFICATION OF A COMMON T-NATURAL-KILLER-CELL PROGENITOR IN HUMAN FETAL THYMUS. JOURNAL OF EXPERIMENTAL MEDICINE, 180(2), 569–576.
- LYMPHOID AND MYELOID DIFFERENTIATION OF FETAL LIVER CD34+ LINEAGE(-) CELLS IN HUMAN THYMIC ORGAN-CULTURE
  Barcena, A., Galy, A. H. M., PUNNONEN, J., Muench, M. O., SCHOLS, D., Roncarolo, M. G., … Spits, H. (1994). LYMPHOID AND MYELOID DIFFERENTIATION OF FETAL LIVER CD34+ LINEAGE(-) CELLS IN HUMAN THYMIC ORGAN-CULTURE. JOURNAL OF EXPERIMENTAL MEDICINE, 180(1), 123–132.
- EXPRESSION OF CD33, CD38, AND HLA-DR ON CD34+ HUMAN FETAL LIVER PROGENITORS WITH A HIGH PROLIFERATIVE POTENTIAL
  Muench, M. O., Cupp, J., Polakoff, J., & Roncarolo, M. G. (1994). EXPRESSION OF CD33, CD38, AND HLA-DR ON CD34+ HUMAN FETAL LIVER PROGENITORS WITH A HIGH PROLIFERATIVE POTENTIAL. BLOOD, 83(11), 3170–3181.
- IL-2 REVERSES HUMAN T-CELL UNRESPONSIVENESS INDUCED BY THYMIC EPITHELIUM IN SCID-HU MICE
  SCHOLS, D., Vandekerckhove, B., Jones, D., & Roncarolo, M. G. (1994). IL-2 REVERSES HUMAN T-CELL UNRESPONSIVENESS INDUCED BY THYMIC EPITHELIUM IN SCID-HU MICE. JOURNAL OF IMMUNOLOGY, 152(5), 2198–2206.
- IN-VIVO CYTOKINE EXPRESSION IN THE THYMUS CD3(HIGH) HUMAN THYMOCYTES ARE ACTIVATED AND ALREADY FUNCTIONALLY DIFFERENTIATED IN HELPER AND CYTOTOXIC-CELLS
  VANDEKERCKHOVE, B. A. E., Barcena, A., SCHOLS, D., MOHANPETERSON, S., Spits, H., & Roncarolo, M. G. (1994). IN-VIVO CYTOKINE EXPRESSION IN THE THYMUS CD3(HIGH) HUMAN THYMOCYTES ARE ACTIVATED AND ALREADY FUNCTIONALLY DIFFERENTIATED IN HELPER AND CYTOTOXIC-CELLS. JOURNAL OF IMMUNOLOGY, 152(4), 1738–1743.
- HIGH-LEVELS OF INTERLEUKIN-10 PRODUCTION IN-VIVO ARE ASSOCIATED WITH TOLERANCE IN SCID PATIENTS TRANSPLANTED WITH HLA MISMATCHED HEMATOPOIETIC STEM-CELLS
  Bacchetta, R., Bigler, M., TOURAINE, J. L., Parkman, R., Tovo, P. A., Abrams, J., … Roncarolo, M. G. (1994). HIGH-LEVELS OF INTERLEUKIN-10 PRODUCTION IN-VIVO ARE ASSOCIATED WITH TOLERANCE IN SCID PATIENTS TRANSPLANTED WITH HLA MISMATCHED HEMATOPOIETIC STEM-CELLS. JOURNAL OF EXPERIMENTAL MEDICINE, 179(2), 493–502.
- IN SEARCH OF T-CELL PROGENITORS IN THE HUMAN FETAL LIVER
  Barcena, A., Muench, M. O., Roncarolo, M. G., & Spits, H. (1994). IN SEARCH OF T-CELL PROGENITORS IN THE HUMAN FETAL LIVER. RESEARCH IN IMMUNOLOGY, 145(2), 120–123.
- IMMUNE-RESPONSES BY CORD-BLOOD CELLS
  Roncarolo, M. G., Bigler, M., CIUTI, E., Martino, S., & Tovo, P. A. (1994). IMMUNE-RESPONSES BY CORD-BLOOD CELLS. BLOOD CELLS, 20(2-3), 573–586.
- PHENOTYPIC AND FUNCTIONAL-ANALYSIS OF T-CELL PRECURSORS IN THE HUMAN FETAL LIVER AND THYMUS - CD7 EXPRESSION IN THE EARLY STAGES OF T-CELL AND MYELOID-CELL DEVELOPMENT
  Barcena, A., Muench, M. O., Galy, A. H. M., Cupp, J., Roncarolo, M. G., Phillips, J. H., & Spits, H. (1993). PHENOTYPIC AND FUNCTIONAL-ANALYSIS OF T-CELL PRECURSORS IN THE HUMAN FETAL LIVER AND THYMUS - CD7 EXPRESSION IN THE EARLY STAGES OF T-CELL AND MYELOID-CELL DEVELOPMENT. BLOOD, 82(11), 3401–3414.
- T-LYMPHOCYTES FROM HUMAN CHIMERAS DO RECOGNIZE ANTIGEN IN THE CONTEXT OF ALLOGENEIC DETERMINANTS OF THE MAJOR HISTOCOMPATIBILITY COMPLEX
  TOURAINE, J. L., Roncarolo, M. G., Plotnicky, H., BACHETTA, R., & Spits, H. (1993). T-LYMPHOCYTES FROM HUMAN CHIMERAS DO RECOGNIZE ANTIGEN IN THE CONTEXT OF ALLOGENEIC DETERMINANTS OF THE MAJOR HISTOCOMPATIBILITY COMPLEX. IMMUNOLOGY LETTERS, 39(1), 9–12.
- HUMAN IG PRODUCTION AND ISOTYPE SWITCHING IN SEVERE COMBINED IMMUNODEFICIENT-HUMAN MICE
  VANDEKERCKHOVE, B. A. E., Jones, D., PUNNONEN, J., SCHOLS, D., Lin, H. C., Duncan, B., … Roncarolo, M. G. (1993). HUMAN IG PRODUCTION AND ISOTYPE SWITCHING IN SEVERE COMBINED IMMUNODEFICIENT-HUMAN MICE. JOURNAL OF IMMUNOLOGY, 151(1), 128–137.
- INDUCING AND ENHANCING EFFECTS OF IL-3, IL-5, AND IL-6 AND GM-CSF ON HISTAMINE-RELEASE FROM HUMAN BASOPHILS
  Miadonna, A., Roncarolo, M. G., Lorini, M., & Tedeschi, A. (1993). INDUCING AND ENHANCING EFFECTS OF IL-3, IL-5, AND IL-6 AND GM-CSF ON HISTAMINE-RELEASE FROM HUMAN BASOPHILS. CLINICAL IMMUNOLOGY AND IMMUNOPATHOLOGY, 67(3), 210–215.
- BACTERIAL SUPERANTIGENS MEDIATE T-CELL DELETIONS IN THE MOUSE SEVERE COMBINED IMMUNODEFICIENCY HUMAN LIVER THYMUS MODEL
  Baccala, R., VANDEKERCKHOVE, B. A. E., Jones, D., Kono, D. H., Roncarolo, M. G., & Theofilopoulos, A. N. (1993). BACTERIAL SUPERANTIGENS MEDIATE T-CELL DELETIONS IN THE MOUSE SEVERE COMBINED IMMUNODEFICIENCY HUMAN LIVER THYMUS MODEL. JOURNAL OF EXPERIMENTAL MEDICINE, 177(5), 1481–1485.
- CHIMERISM AND TOLERANCE TO HOST AND DONOR IN SEVERE COMBINED IMMUNODEFICIENCIES TRANSPLANTED WITH FETAL LIVER STEM-CELLS
  Bacchetta, R., VANDEKERCKHOVE, B. A. E., TOURAINE, J. L., Bigler, M., Martino, S., Gebuhrer, L., … Roncarolo, M. G. (1993). CHIMERISM AND TOLERANCE TO HOST AND DONOR IN SEVERE COMBINED IMMUNODEFICIENCIES TRANSPLANTED WITH FETAL LIVER STEM-CELLS. JOURNAL OF CLINICAL INVESTIGATION, 91(3), 1067–1078.
- FETAL LIVER-TRANSPLANTATION - BIOLOGY AND CLINICAL-RESULTS
  TOURAINE, J. L., Roncarolo, M. G., Bacchetta, R., Raudrant, D., Rebaud, A., Laplace, S., … Vullo, C. (1993). FETAL LIVER-TRANSPLANTATION - BIOLOGY AND CLINICAL-RESULTS. Presented at the INTERNATIONAL SYMP ON BONE MARROW TRANSPLANTATION FROM ALTERNATIVE DONORS : BIOLOGY, CLINICAL RESULTS, REGISTRY ACTIVITIES, PAVIA,ITALY: NATURE PUBLISHING GROUP.
- THYMIC SELECTION OF THE HUMAN T-CELL RECEPTOR V-BETA REPERTOIRE IN SCID-HU MICE
  VANDEKERCKHOVE, B. A. E., Baccala, R., Jones, D., Kono, D. H., Theofilopoulos, A. N., & Roncarolo, M. G. (1992). THYMIC SELECTION OF THE HUMAN T-CELL RECEPTOR V-BETA REPERTOIRE IN SCID-HU MICE. JOURNAL OF EXPERIMENTAL MEDICINE, 176(6), 1619–1624.
- INTERLEUKIN-10 INHIBITS ALLOGENEIC PROLIFERATIVE AND CYTOTOXIC T-CELL RESPONSES GENERATED IN PRIMARY MIXED LYMPHOCYTE-CULTURES
  Bejarano, M. T., Malefyt, R. D., Abrams, J. S., Bigler, M., Bacchetta, R., deVries, J. E., & Roncarolo, M. G. (1992). INTERLEUKIN-10 INHIBITS ALLOGENEIC PROLIFERATIVE AND CYTOTOXIC T-CELL RESPONSES GENERATED IN PRIMARY MIXED LYMPHOCYTE-CULTURES. INTERNATIONAL IMMUNOLOGY, 4(12), 1389–1397.
- INTERLEUKIN-10
  Malefyt, R. D., Yssel, H., Roncarolo, M. G., Spits, H., & deVries, J. E. (1992). INTERLEUKIN-10. CURRENT OPINION IN IMMUNOLOGY, 4(3), 314–320.
- STRATEGIES OF ANTICYTOKINE MONOCLONAL-ANTIBODY DEVELOPMENT - IMMUNOASSAY OF IL-10 AND IL-5 IN CLINICAL-SAMPLES
  Abrams, J. S., Roncarolo, M. G., Yssel, H., Andersson, U., Gleich, G. J., & SILVER, J. E. (1992). STRATEGIES OF ANTICYTOKINE MONOCLONAL-ANTIBODY DEVELOPMENT - IMMUNOASSAY OF IL-10 AND IL-5 IN CLINICAL-SAMPLES. IMMUNOLOGICAL REVIEWS, 127, 5–24.
- INDUCTION OF ISOTYPE SWITCHING AND IG PRODUCTION BY CD5+ AND CD10+ HUMAN FETAL B-CELLS
  PUNNONEN, J., AVERSA, G. G., Vandekerckhove, B., Roncarolo, M. G., & deVries, J. E. (1992). INDUCTION OF ISOTYPE SWITCHING AND IG PRODUCTION BY CD5+ AND CD10+ HUMAN FETAL B-CELLS. JOURNAL OF IMMUNOLOGY, 148(11), 3398–3404.
- MEMBRANES OF ACTIVATED CD4+ T-CELLS EXPRESSING T-CELL RECEPTOR (TCR) ALPHA-BETA OR TCR GAMMA-DELTA INDUCE IGE SYNTHESIS BY HUMAN B-CELLS IN THE PRESENCE OF INTERLEUKIN-4
  Gascan, H., AVERSA, G. G., Gauchat, J. F., VANVLASSELAER, P., Roncarolo, M. G., Yssel, H., … deVries, J. E. (1992). MEMBRANES OF ACTIVATED CD4+ T-CELLS EXPRESSING T-CELL RECEPTOR (TCR) ALPHA-BETA OR TCR GAMMA-DELTA INDUCE IGE SYNTHESIS BY HUMAN B-CELLS IN THE PRESENCE OF INTERLEUKIN-4. EUROPEAN JOURNAL OF IMMUNOLOGY, 22(5), 1133–1141.
- HUMAN HEMATOPOIETIC-CELLS AND THYMIC EPITHELIAL-CELLS INDUCE TOLERANCE VIA DIFFERENT MECHANISMS IN THE SCID-HU MOUSE THYMUS
  VANDEKERCKHOVE, B. A. E., Namikawa, R., Bacchetta, R., & Roncarolo, M. G. (1992). HUMAN HEMATOPOIETIC-CELLS AND THYMIC EPITHELIAL-CELLS INDUCE TOLERANCE VIA DIFFERENT MECHANISMS IN THE SCID-HU MOUSE THYMUS. JOURNAL OF EXPERIMENTAL MEDICINE, 175(4), 1033–1043.
- SCID-HU MICE AS A MODEL TO STUDY TOLERANCE AFTER FETAL STEM-CELL TRANSPLANTATION
  Roncarolo, M. G., & Vandekerckhove, B. (1992). SCID-HU MICE AS A MODEL TO STUDY TOLERANCE AFTER FETAL STEM-CELL TRANSPLANTATION. Presented at the SYMP ON FOETAL AND NEONATAL CELL TRANSPLANTATION AND RETROVIRAL GENE THERAPY, ANNECY,FRANCE: STOCKTON PRESS.
- T-CELL REPERTOIRE AND TOLERANCE AFTER FETAL STEM-CELL TRANSPLANTATION
  Roncarolo, M. G., & Bacchetta, R. (1992). T-CELL REPERTOIRE AND TOLERANCE AFTER FETAL STEM-CELL TRANSPLANTATION. Presented at the SYMP ON FOETAL AND NEONATAL CELL TRANSPLANTATION AND RETROVIRAL GENE THERAPY, ANNECY,FRANCE: STOCKTON PRESS.
- INUTERO TRANSPLANTATION OF STEM-CELLS IN HUMANS - IMMUNOLOGICAL ASPECTS AND CLINICAL FOLLOW-UP OF PATIENTS
  TOURAINE, J. L., Raudrant, D., Rebaud, A., Roncarolo, M. G., Laplace, S., Gebuhrer, L., … Vullo, C. (1992). INUTERO TRANSPLANTATION OF STEM-CELLS IN HUMANS - IMMUNOLOGICAL ASPECTS AND CLINICAL FOLLOW-UP OF PATIENTS. Presented at the SYMP ON FOETAL AND NEONATAL CELL TRANSPLANTATION AND RETROVIRAL GENE THERAPY, ANNECY,FRANCE: STOCKTON PRESS.
- RECIPROCAL HYBRID JOINTS DEMONSTRATE SUCCESSIVE V-J REARRANGEMENTS ON THE SAME CHROMOSOME IN THE HUMAN TCR GAMMA LOCUS
  Alexandre, D., Chuchana, P., Roncarolo, M. G., Yssel, H., Spits, H., Lefranc, G., & Lefranc, M. P. (1991). RECIPROCAL HYBRID JOINTS DEMONSTRATE SUCCESSIVE V-J REARRANGEMENTS ON THE SAME CHROMOSOME IN THE HUMAN TCR GAMMA LOCUS. INTERNATIONAL IMMUNOLOGY, 3(10), 973–982.
- INTERLEUKIN-10 (IL-10) AND VIRAL-IL-10 STRONGLY REDUCE ANTIGEN-SPECIFIC HUMAN T-CELL PROLIFERATION BY DIMINISHING THE ANTIGEN-PRESENTING CAPACITY OF MONOCYTES VIA DOWN-REGULATION OF CLASS-II MAJOR HISTOCOMPATIBILITY COMPLEX EXPRESSION
  Malefyt, R. D., Haanen, J., Spits, H., Roncarolo, M. G., TEVELDE, A., Figdor, C., … deVries, J. E. (1991). INTERLEUKIN-10 (IL-10) AND VIRAL-IL-10 STRONGLY REDUCE ANTIGEN-SPECIFIC HUMAN T-CELL PROLIFERATION BY DIMINISHING THE ANTIGEN-PRESENTING CAPACITY OF MONOCYTES VIA DOWN-REGULATION OF CLASS-II MAJOR HISTOCOMPATIBILITY COMPLEX EXPRESSION. JOURNAL OF EXPERIMENTAL MEDICINE, 174(4), 915–924.
- NATURAL-KILLER-CELL CLONES CAN EFFICIENTLY PROCESS AND PRESENT PROTEIN ANTIGENS
  Roncarolo, M. G., Bigler, M., HAANEN, J. B. A., Yssel, H., Bacchetta, R., deVries, J. E., & Spits, H. (1991). NATURAL-KILLER-CELL CLONES CAN EFFICIENTLY PROCESS AND PRESENT PROTEIN ANTIGENS. JOURNAL OF IMMUNOLOGY, 147(3), 781–787.
- CLONAL ANALYSIS OF THE PERIPHERAL T-CELL COMPARTMENT OF THE SCID-HU MOUSE
  VANDEKERCKHOVE, B. A. E., Krowka, J. F., McCune, J. M., deVries, J. E., Spits, H., & Roncarolo, M. G. (1991). CLONAL ANALYSIS OF THE PERIPHERAL T-CELL COMPARTMENT OF THE SCID-HU MOUSE. JOURNAL OF IMMUNOLOGY, 146(12), 4173–4179.
- LOW MW B-CELL GROWTH-FACTOR POTENTIATES HISTAMINE-RELEASE FROM HUMAN BASOPHIL LEUKOCYTES
  Tedeschi, A., Roncarolo, M. G., Lorini, M., & Miadonna, A. (1991). LOW MW B-CELL GROWTH-FACTOR POTENTIATES HISTAMINE-RELEASE FROM HUMAN BASOPHIL LEUKOCYTES. IMMUNOLOGY, 73(2), 217–221.
- Cloning of a novel cell type from human fetal liver expressing cytoplasmic CD3 delta and epsilon but not membrane CD3.
  Hori, T., de Waal Malefyt, R., Duncan, B. W., Harrison, M. R., Roncarolo, M. G., & Spits, H. (1991). Cloning of a novel cell type from human fetal liver expressing cytoplasmic CD3 delta and epsilon but not membrane CD3. International Immunology, 3(4), 353–357.
- Regulation of human IgE synthesis: the role of CD4+ and CD8+ T-cells and the inhibitory effects of interferon-alpha.
  Gauchat, J. F., Gascan, H., Roncarolo, M. G., Rousset, F., Pène, J., & de Vries, J. E. (1991). Regulation of human IgE synthesis: the role of CD4+ and CD8+ T-cells and the inhibitory effects of interferon-alpha. The European Respiratory Journal. Supplement, 13, 31s–38s.
- HUMAN B-CELL CLONES CAN BE INDUCED TO PROLIFERATE AND TO SWITCH TO IGE AND IGG4 SYNTHESIS BY INTERLEUKIN-4 AND A SIGNAL PROVIDED BY ACTIVATED CD4+ T-CELL CLONES
  Gascan, H., Gauchat, J. F., Roncarolo, M. G., Yssel, H., Spits, H., & deVries, J. E. (1991). HUMAN B-CELL CLONES CAN BE INDUCED TO PROLIFERATE AND TO SWITCH TO IGE AND IGG4 SYNTHESIS BY INTERLEUKIN-4 AND A SIGNAL PROVIDED BY ACTIVATED CD4+ T-CELL CLONES. JOURNAL OF EXPERIMENTAL MEDICINE, 173(3), 747–750.
- In utero transplantation of hemopoietic stem cells in humans.
  TOURAINE, J. L., Raudrant, D., Royo, C., Rebaud, A., BARBIER, F., Roncarolo, M. G., … Bétuel, H. (1991). In utero transplantation of hemopoietic stem cells in humans. Transplantation Proceedings, 23(1), 1706–1708.
- NEW DEVELOPMENTS IN STEM-CELL TRANSPLANTATION WITH SPECIAL REFERENCE TO THE 1ST INUTERO TRANSPLANTS IN HUMANS
  TOURAINE, J. L., Raudrant, D., Vullo, C., Frappaz, D., Freycon, F., Rebaud, A., … Zabot, M. T. (1991). NEW DEVELOPMENTS IN STEM-CELL TRANSPLANTATION WITH SPECIAL REFERENCE TO THE 1ST INUTERO TRANSPLANTS IN HUMANS. Presented at the INTERNATIONAL COURSE ON BONE MARROW TRANSPLANTATION IN CHILDREN, ERICE,ITALY: STOCKTON PRESS.
- DO HUMAN TH1 AND TH2 CD4+ CLONES EXIST
  deVries, J. E., Malefyt, R. D., Yssel, H., Roncarolo, M. G., & Spits, H. (1991). DO HUMAN TH1 AND TH2 CD4+ CLONES EXIST. RESEARCH IN IMMUNOLOGY, 142(1), 59–63.
- A SCID PATIENT RECONSTITUTED WITH HLA-INCOMPATIBLE FETAL STEM-CELLS AS A MODEL FOR STUDYING TRANSPLANTATION TOLERANCE
  Roncarolo, M. G., Bacchetta, R., Bigler, M., TOURAINE, J. L., deVries, J. E., & Spits, H. (1991). A SCID PATIENT RECONSTITUTED WITH HLA-INCOMPATIBLE FETAL STEM-CELLS AS A MODEL FOR STUDYING TRANSPLANTATION TOLERANCE. BLOOD CELLS, 17(2), 391–402.
- THE CAPACITY OF INTERLEUKIN-4 TO INDUCE INVITRO IGE SYNTHESIS BY B-CELLS OF PATIENTS WITH COMMON VARIABLE IMMUNODEFICIENCY
  Pastorelli, G., Roncarolo, M. G., Peronne, C., Tovo, P. A., & deVries, J. E. (1990). THE CAPACITY OF INTERLEUKIN-4 TO INDUCE INVITRO IGE SYNTHESIS BY B-CELLS OF PATIENTS WITH COMMON VARIABLE IMMUNODEFICIENCY. CLINICAL AND EXPERIMENTAL IMMUNOLOGY, 82(1), 120–127.
- CORD BLOOD B-CELLS ARE MATURE IN THEIR CAPACITY TO SWITCH TO IGE-PRODUCING CELLS IN RESPONSE TO INTERLEUKIN-4 INVITRO
  Pastorelli, G., Rousset, F., Pene, J., Peronne, C., Roncarolo, M. G., Tovo, P. A., & deVries, J. E. (1990). CORD BLOOD B-CELLS ARE MATURE IN THEIR CAPACITY TO SWITCH TO IGE-PRODUCING CELLS IN RESPONSE TO INTERLEUKIN-4 INVITRO. CLINICAL AND EXPERIMENTAL IMMUNOLOGY, 82(1), 114–119.
- PRESENCE OF HOST-REACTIVE AND MHC-RESTRICTED T-CELLS IN A TRANSPLANTED SEVERE COMBINED IMMUNODEFICIENT (SCID) PATIENT SUGGEST POSITIVE SELECTION AND ABSENCE OF CLONAL DELETION
  Spits, H., TOURAINE, J. L., Yssel, H., deVries, J. E., & Roncarolo, M. G. (1990). PRESENCE OF HOST-REACTIVE AND MHC-RESTRICTED T-CELLS IN A TRANSPLANTED SEVERE COMBINED IMMUNODEFICIENT (SCID) PATIENT SUGGEST POSITIVE SELECTION AND ABSENCE OF CLONAL DELETION. IMMUNOLOGICAL REVIEWS, 116, 101–116.
- HOST-REACTIVE CD4+ AND CD8+ T-CELL CLONES ISOLATED FROM A HUMAN CHIMERA PRODUCE IL-5, IL-2, IFN-GAMMA- AND GRANULOCYTE MACROPHAGE-COLONY-STIMULATING FACTOR BUT NOT IL-4
  Bacchetta, R., MALEFIJT, R. D., Yssel, H., Abrams, J., deVries, J. E., Spits, H., & Roncarolo, M. G. (1990). HOST-REACTIVE CD4+ AND CD8+ T-CELL CLONES ISOLATED FROM A HUMAN CHIMERA PRODUCE IL-5, IL-2, IFN-GAMMA- AND GRANULOCYTE MACROPHAGE-COLONY-STIMULATING FACTOR BUT NOT IL-4. JOURNAL OF IMMUNOLOGY, 144(3), 902–908.
- INTERLEUKIN-4 SUPPRESSES IMMUNOGLOBULIN PRODUCTION BY PERIPHERAL-BLOOD LYMPHOCYTES OF PATIENTS WITH COMMON VARIABLE IMMUNODEFICIENCY (CVI) INDUCED BY SUPERNATANTS OF T-CELL CLONES
  Pastorelli, G., Roncarolo, M. G., TOURAINE, J. L., Rousset, F., Pene, J., & deVries, J. E. (1989). INTERLEUKIN-4 SUPPRESSES IMMUNOGLOBULIN PRODUCTION BY PERIPHERAL-BLOOD LYMPHOCYTES OF PATIENTS WITH COMMON VARIABLE IMMUNODEFICIENCY (CVI) INDUCED BY SUPERNATANTS OF T-CELL CLONES. CLINICAL AND EXPERIMENTAL IMMUNOLOGY, 78(3), 341–347.
- PERIPHERAL-BLOOD LYMPHOCYTES OF PATIENTS WITH COMMON VARIABLE IMMUNODEFICIENCY (CVI) PRODUCE REDUCED LEVELS OF INTERLEUKIN-4, INTERLEUKIN-2 AND INTERFERON-GAMMA, BUT PROLIFERATE NORMALLY UPON ACTIVATION BY MITOGENS
  Pastorelli, G., Roncarolo, M. G., TOURAINE, J. L., PERONNE, G., Tovo, P. A., & deVries, J. E. (1989). PERIPHERAL-BLOOD LYMPHOCYTES OF PATIENTS WITH COMMON VARIABLE IMMUNODEFICIENCY (CVI) PRODUCE REDUCED LEVELS OF INTERLEUKIN-4, INTERLEUKIN-2 AND INTERFERON-GAMMA, BUT PROLIFERATE NORMALLY UPON ACTIVATION BY MITOGENS. CLINICAL AND EXPERIMENTAL IMMUNOLOGY, 78(3), 334–340.
- INUTERO TRANSPLANTATION OF STEM-CELLS IN BARE LYMPHOCYTE SYNDROME
  TOURAINE, J. L., Raudrant, D., Royo, C., Rebaud, A., Roncarolo, M. G., Souillet, G., … Betuel, H. (1989). INUTERO TRANSPLANTATION OF STEM-CELLS IN BARE LYMPHOCYTE SYNDROME. LANCET, 1(8651), 1382–1382.
- [Phenotype expression and production of IL-2 by tonsillar and blood lymphocytes in patients with tonsil pathology].
  Bussi, M., Carlevato, M. T., ZOPPO, M., & Roncarolo, M. G. (1989). [Phenotype expression and production of IL-2 by tonsillar and blood lymphocytes in patients with tonsil pathology]. Acta Otorhinolaryngologica Italica , 9(2), 149–159.
- UNMATCHED STEM-CELL TRANSPLANTATION AS A POSSIBLE ALTERNATIVE TO BONE-MARROW TRANSPLANTATION
  TOURAINE, J. L., Royo, C., Roncarolo, M. G., Murray, K., & DEBOUTEILLER, O. (1989). UNMATCHED STEM-CELL TRANSPLANTATION AS A POSSIBLE ALTERNATIVE TO BONE-MARROW TRANSPLANTATION. TRANSPLANTATION PROCEEDINGS, 21(1), 3112–3113.
- ANTIGEN RECOGNITION BY MHC-INCOMPATIBLE CELLS OF A HUMAN MISMATCHED CHIMERA
  Roncarolo, M. G., Yssel, H., TOURAINE, J. L., Bacchetta, R., Gebuhrer, L., deVries, J. E., & Spits, H. (1988). ANTIGEN RECOGNITION BY MHC-INCOMPATIBLE CELLS OF A HUMAN MISMATCHED CHIMERA. JOURNAL OF EXPERIMENTAL MEDICINE, 168(6), 2139–2152.
- GENOMIC ORGANIZATION OF THE HUMAN T-CELL ANTIGEN-RECEPTOR ALPHA,SIGMA LOCUS
  Satyanarayana, K., Hata, S., Devlin, P., Roncarolo, M. G., deVries, J. E., Spits, H., … Krangel, M. S. (1988). GENOMIC ORGANIZATION OF THE HUMAN T-CELL ANTIGEN-RECEPTOR ALPHA,SIGMA LOCUS. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 85(21), 8166–8170.
- INTERLEUKIN-2 PRODUCTION AND INTERLEUKIN-2 RECEPTOR EXPRESSION IN CHILDREN WITH NEWLY DIAGNOSED DIABETES
  Roncarolo, M. G., ZOPPO, M., Bacchetta, R., Gabiano, C., Sacchetti, C., Cerutti, F., & Tovo, P. A. (1988). INTERLEUKIN-2 PRODUCTION AND INTERLEUKIN-2 RECEPTOR EXPRESSION IN CHILDREN WITH NEWLY DIAGNOSED DIABETES. CLINICAL IMMUNOLOGY AND IMMUNOPATHOLOGY, 49(1), 53–62.
- AUTOREACTIVE T-CELL CLONES SPECIFIC FOR CLASS-I AND CLASS-II HLA ANTIGENS ISOLATED FROM A HUMAN CHIMERA
  Roncarolo, M. G., Yssel, H., TOURAINE, J. L., Betuel, H., deVries, J. E., & Spits, H. (1988). AUTOREACTIVE T-CELL CLONES SPECIFIC FOR CLASS-I AND CLASS-II HLA ANTIGENS ISOLATED FROM A HUMAN CHIMERA. JOURNAL OF EXPERIMENTAL MEDICINE, 167(5), 1523–1534.
- FETAL TISSUE-TRANSPLANTATION, BONE-MARROW TRANSPLANTATION AND PROSPECTIVE GENE-THERAPY IN SEVERE IMMUNODEFICIENCIES AND ENZYME DEFICIENCIES
  TOURAINE, J. L., Roncarolo, M. G., Royo, C., & Touraine, F. (1987). FETAL TISSUE-TRANSPLANTATION, BONE-MARROW TRANSPLANTATION AND PROSPECTIVE GENE-THERAPY IN SEVERE IMMUNODEFICIENCIES AND ENZYME DEFICIENCIES. THYMUS, 10(1-2), 75–87.
- COOPERATION BETWEEN MAJOR HISTOCOMPATIBILITY COMPLEX MISMATCHED MONONUCLEAR-CELLS FROM A HUMAN CHIMERA IN THE PRODUCTION OF ANTIGEN-SPECIFIC ANTIBODY
  Roncarolo, M. G., TOURAINE, J. L., & Banchereau, J. (1986). COOPERATION BETWEEN MAJOR HISTOCOMPATIBILITY COMPLEX MISMATCHED MONONUCLEAR-CELLS FROM A HUMAN CHIMERA IN THE PRODUCTION OF ANTIGEN-SPECIFIC ANTIBODY. JOURNAL OF CLINICAL INVESTIGATION, 77(3), 673–680.
- STUDIES OF EBV-LYMPHOID CELL-INTERACTIONS IN 2 PATIENTS WITH THE X-LINKED LYMPHOPROLIFERATIVE SYNDROME - NORMAL EBV-SPECIFIC HLA-RESTRICTED CYTOTOXICITY
  Rousset, F., Souillet, G., Roncarolo, M. G., & LAMELIN, J. P. (1986). STUDIES OF EBV-LYMPHOID CELL-INTERACTIONS IN 2 PATIENTS WITH THE X-LINKED LYMPHOPROLIFERATIVE SYNDROME - NORMAL EBV-SPECIFIC HLA-RESTRICTED CYTOTOXICITY. CLINICAL AND EXPERIMENTAL IMMUNOLOGY, 63(2), 280–289.
- Fetal liver transplantation in immunodeficiencies and inborn errors of metabolism.
  TOURAINE, J. L., Roncarolo, M. G., Marseglia, G. L., Souillet, G., Bétend, B., Bétuel, H., … François, R. (1985). Fetal liver transplantation in immunodeficiencies and inborn errors of metabolism. Progress in Clinical and Biological Research, 193, 299–313.
- TOLERANCE TO ALLOANTIGENS AND RECOGNITION FOR ALLO+X INDUCED IN HUMANS BY FETAL STEM CELL TRANSPLANTATION
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- First Occurrence of Plasmablastic Lymphoma in Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Disease Patient and Review of the Literature
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- Engineered T Regulatory Type 1 Cells for Clinical Application
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- Gene Therapy for Adenosine Deaminase Deficiency: A Comprehensive Evaluation of Short- and Medium-Term Safety
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- Genome Editing for IL-10 Deficiency in Purified Hematopoietic Stem Cells
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- CRISPR-Based Therapy for IPEX Syndrome as a Model of Genetic Autoimmunity
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- Reprogramming human T cell function and specificity with non-viral genome targeting
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- Tregopathies: Monogenic diseases resulting in regulatory T-cell deficiency.
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- Lentiviral Gene Therapy in HSCs Restores Lineage-Specific Foxp3 Expression and Suppresses Autoimmunity in a Mouse Model of IPEX Syndrome.
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- Engineering Regenerative Thymic Tissues to Restore Long-Term T Cell Lymphopoiesis
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- IL-10-Engineered Human CD4(+) Tr1 Cells Eliminate Myeloid Leukemia in an HLA Class I-Dependent Mechanism
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- LV.InsulinB9-23/Anti-CD3 mAb Inhibits Recurrence of Autoimmunity in Diabetic NOD Mice After Islet Transplant
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- Insulin B9-23 LV-Driven Expression in Hepatocytes Combined With Suboptimal Dose of Anti-CD3 mAb Cures Type 1 Diabetes in NOD Mice
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- Gene correction for SCID-X1 in long-term hematopoietic stem cells.
  Pavel-Dinu, M., Wiebking, V., Dejene, B. T., Srifa, W., Mantri, S., Nicolas, C. E., … Porteus, M. H. (2019). Gene correction for SCID-X1 in long-term hematopoietic stem cells. Nature Communications, 10(1), 1634.
- Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study.
  Ferrua, F., Cicalese, M. P., Galimberti, S., Giannelli, S., Dionisio, F., Barzaghi, F., … Aiuti, A. (2019). Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study. The Lancet. Haematology.
- APVO210: A Bispecific Anti-CD86-IL-10 Fusion Protein (ADAPTIR™) to Induce Antigen-Specific T Regulatory Type 1 Cells.
  Pellerin, L., Chen, P., Gregori, S., Hernandez-Hoyos, G., Bacchetta, R., & Roncarolo, M. G. (2018). APVO210: A Bispecific Anti-CD86-IL-10 Fusion Protein (ADAPTIR™) to Induce Antigen-Specific T Regulatory Type 1 Cells. Frontiers in Immunology, 9, 881.
- Driving Medical Innovation Through Interdisciplinarity: Unique Opportunities and Challenges.
  Gohar, F., Maschmeyer, P., Mfarrej, B., Lemaire, M., Wedderburn, L. R., Roncarolo, M. G., & van Royen-Kerkhof, A. (2019). Driving Medical Innovation Through Interdisciplinarity: Unique Opportunities and Challenges. Frontiers in Medicine, 6, 35.
- Author Correction: Gene correction for SCID-X1 in long-term hematopoietic stem cells.
  Pavel-Dinu, M., Wiebking, V., Dejene, B. T., Srifa, W., Mantri, S., Nicolas, C. E., … Porteus, M. H. (2019). Author Correction: Gene correction for SCID-X1 in long-term hematopoietic stem cells. Nature Communications, 10(1), 2021.
- Immunoregulatory Cell Therapy with Lentiviral-Mediated FOXP3 Converted CD4+T Cells into Treg Cells: Towards the Proof-of-Concept Application in IPEX Syndrome
  Sato, Y., Passerini, L., Roncarolo, M.-G., & Bacchetta, R. (2019). Immunoregulatory Cell Therapy with Lentiviral-Mediated FOXP3 Converted CD4+T Cells into Treg Cells: Towards the Proof-of-Concept Application in IPEX Syndrome. MOLECULAR THERAPY, 27(4), 311.
- Gene correction for SCID-X1 in long-term hematopoietic stem cells (vol 10, 1634, 2019)
  Pavel-Dinu, M., Wiebking, V., Dejene, B. T., Srifa, W., Mantri, S., Nicolas, C. E., … Porteus, M. H. (2019). Gene correction for SCID-X1 in long-term hematopoietic stem cells (vol 10, 1634, 2019). NATURE COMMUNICATIONS, 10.
- Gene correction for SCID-X1 in long-term hematopoietic stem cells
  Pavel-Dinu, M., Wiebking, V., Dejene, B. T., Srifa, W., Mantri, S., Nicolas, C. E., … Porteus, M. H. (2019). Gene correction for SCID-X1 in long-term hematopoietic stem cells. NATURE COMMUNICATIONS, 10.
- Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study
  Ferrua, F., Cicalese, M. P., Galimberti, S., Giannelli, S., Dionisio, F., Barzaghi, F., … Aiuti, A. (2019). Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study. LANCET HAEMATOLOGY, 6(5), E239–E253.
- Gene therapy for primary immunodeficiency.
  Booth, C., Romano, R., Roncarolo, M. G., & Thrasher, A. J. (2019). Gene therapy for primary immunodeficiency. Human Molecular Genetics.
- Graft Engineering and Adoptive Immunotherapy: New Approaches to Promote Immune Tolerance After Hematopoietic Stem Cell Transplantation
  Bertaina, A., & Roncarolo, M. G. (2019). Graft Engineering and Adoptive Immunotherapy: New Approaches to Promote Immune Tolerance After Hematopoietic Stem Cell Transplantation. FRONTIERS IN IMMUNOLOGY, 10.
- Lentiviral Gene Therapy in HSCs Restores Lineage-Specific Foxp3 Expression and Suppresses Autoimmunity in a Mouse Model of IPEX Syndrome
  Masiuk, K. E., Laborada, J., Roncarolo, M. G., Hollis, R. P., & Kohn, D. B. (2019). Lentiviral Gene Therapy in HSCs Restores Lineage-Specific Foxp3 Expression and Suppresses Autoimmunity in a Mouse Model of IPEX Syndrome. CELL STEM CELL, 24(2), 309-+.
- Author Correction: Gene correction for SCID-X1 in long-term hematopoietic stem cells.
  Pavel-Dinu, M., Wiebking, V., Dejene, B. T., Srifa, W., Mantri, S., Nicolas, C. E., … Porteus, M. H. (2019). Author Correction: Gene correction for SCID-X1 in long-term hematopoietic stem cells. Nature Communications, 10(1), 5624.
- A beta T-Cell/CD19 B-Cell Depleted Haploidentical Stem Cell Transplantation: A New Platform for Curing Rare and Monogenic Disorders
  Bertaina, A., Bacchetta, R., Lewis, D. B., Grimm, P. C., Shah, A. J., Agarwal, R., … Roncarolo, M. G. (2020). A beta T-Cell/CD19 B-Cell Depleted Haploidentical Stem Cell Transplantation: A New Platform for Curing Rare and Monogenic Disorders. BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 26(3), S288.
- Early Epigenetic Immune Quantification Following Alpha/Beta T-Cell/CD19 B-Cell Depleted Haploidentical Stem Cell Transplant Correlates with CD4+T Cell Recovery at Day+100
  Mayers, M., Schulze, J., Barbarito, G., Lakshmanan, U., Parkman, R., Weinberg, K. I., … Bertaina, A. (2020). Early Epigenetic Immune Quantification Following Alpha/Beta T-Cell/CD19 B-Cell Depleted Haploidentical Stem Cell Transplant Correlates with CD4+T Cell Recovery at Day+100. BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 26(3), S305.
- Genome editing of donor-derived T-cells to generate allogenic chimeric antigen receptor-modified T cells: Optimizing aß T cell-depleted haploidentical hematopoietic stem cell transplantation.
  Wiebking, V., Lee, C. M., Mostrel, N., Lahiri, P., Bak, R., Bao, G., … Porteus, M. H. (2020). Genome editing of donor-derived T-cells to generate allogenic chimeric antigen receptor-modified T cells: Optimizing aß T cell-depleted haploidentical hematopoietic stem cell transplantation. Haematologica.
- Gene Therapy for Wiskott-Aldrich Syndrome: History, New Vectors, Future Directions.
  Ferrua, F., Marangoni, F., Aiuti, A., & Roncarolo, M. G. (2020). Gene Therapy for Wiskott-Aldrich Syndrome: History, New Vectors, Future Directions. The Journal of Allergy and Clinical Immunology.
- Engineered type 1 regulatory T cells designed for clinical use kill primary pediatric acute myeloid leukemia cells
  Cieniewicz, B., Uyeda, M. J., Chen, P. P., Sayitoglu, E. C., Liu, J. M.-H., Andolfi, G., … Roncarolo, M. G. (2020). Engineered type 1 regulatory T cells designed for clinical use kill primary pediatric acute myeloid leukemia cells. Haematologica.
- Celebrating 20 years of FOCIS.
  Roncarolo, M. G., & Anderson, M. S. (2020). Celebrating 20 years of FOCIS. Science Immunology, 5(52).
- InsB9-23 Gene Transfer To Hepatocytes-Based Combined Therapy Abrogates Recurrence of Type-1 Diabetes After Islet Transplantation.
  Russo, F., Citro, A., Squeri, G., Sanvito, F., Monti, P., Gregori, S., … Annoni, A. (2020). InsB9-23 Gene Transfer To Hepatocytes-Based Combined Therapy Abrogates Recurrence of Type-1 Diabetes After Islet Transplantation. Diabetes.
- Alloantigen-specific Tr1 cells designed to prevent GvHD have a distinct molecular identity and suppress through CTLA-4 and PD-1
  Cepika, A.-M., Chen, P. P., Uyeda, M. J., Cieniewicz, B., Narula, M., Amaya, L., … Roncarolo, M. G. (2020). Alloantigen-specific Tr1 cells designed to prevent GvHD have a distinct molecular identity and suppress through CTLA-4 and PD-1. ournal for ImmunoTherapy of Cancer.
- Human-engineered Treg-like cells suppress FOXP3-deficient T cells but preserve adaptive immune responses in vivo.
  Sato, Y., Passerini, L., Piening, B. D., Uyeda, M. J., Goodwin, M., Gregori, S., … Bacchetta, R. (2020). Human-engineered Treg-like cells suppress FOXP3-deficient T cells but preserve adaptive immune responses in vivo. Clinical & Translational Immunology, 9(11), e1214.
- Hergen Spits-A legend at the top of his career.
  Mjosberg, J., Roncarolo, M. G., & Blom, B. (2021). Hergen Spits-A legend at the top of his career. Allergy.
- Development of beta-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease.
  Lattanzi, A., Camarena, J., Lahiri, P., Segal, H., Srifa, W., Vakulskas, C. A., … Porteus, M. H. (2021). Development of beta-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease. Science Translational Medicine, 13(598).
- The Yin and Yang of Type 1 Regulatory T Cells: From Discovery to Clinical Application.
  Sayitoglu, E. C., Freeborn, R. A., & Roncarolo, M. G. (2021). The Yin and Yang of Type 1 Regulatory T Cells: From Discovery to Clinical Application. Frontiers in Immunology, 12, 693105.
- BHLHE40 Regulates IL-10 and IFN-? Production in T Cells but Does Not Interfere With Human Type 1 Regulatory T Cell Differentiation
  Uyeda, M. J., Freeborn, R. A., Cieniewicz, B., Romano, R., Chen, P. P., Liu, J. M.-H., … Roncarolo, M. G. (2021). BHLHE40 Regulates IL-10 and IFN-? Production in T Cells but Does Not Interfere With Human Type 1 Regulatory T Cell Differentiation. Frontiers in Immunology.
- Pre-clinical development and molecular characterization of an engineered type 1 regulatory T-cell product suitable for immunotherapy.
  Liu, J. M.-H., Chen, P., Uyeda, M. J., Cieniewicz, B., Sayitoglu, E. C., Thomas, B. C., … Roncarolo, M. G. (2021). Pre-clinical development and molecular characterization of an engineered type 1 regulatory T-cell product suitable for immunotherapy. Cytotherapy.
- Alloantigen-specific type 1 regulatory T cells suppress through CTLA-4 and PD-1 pathways and persist long-term in patients.
  Chen, P. P., Cepika, A.-M., Agarwal-Hashmi, R., Saini, G., Uyeda, M. J., Louis, D. M., … Roncarolo, M. G. (2021). Alloantigen-specific type 1 regulatory T cells suppress through CTLA-4 and PD-1 pathways and persist long-term in patients. Science Translational Medicine, 13(617), eabf5264.
- Co-Expression of FOXP3FL and FOXP3?2 Isoforms Is Required for Optimal Treg-Like Cell Phenotypes and Suppressive Function.
  Sato, Y., Liu, J., Lee, E., Perriman, R., Roncarolo, M. G., & Bacchetta, R. (2021). Co-Expression of FOXP3FL and FOXP3?2 Isoforms Is Required for Optimal Treg-Like Cell Phenotypes and Suppressive Function. Frontiers in Immunology, 12, 752394.
- Downregulation of SATB1 by miRNAs Reduces Megakaryocyte/Erythroid Progenitor Expansion in pre-clinical models of Diamond Blackfan Anemia
  Wilkes, M. C., Scanlon, V., Shibuya, A., Cepika, A.-M., Eskin, A., Chen, Z., … Sakamoto, K. M. (2022). Downregulation of SATB1 by miRNAs Reduces Megakaryocyte/Erythroid Progenitor Expansion in pre-clinical models of Diamond Blackfan Anemia. Experimental Hematology.

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LPCH- Stem Cell Transplant (Palo Alto)

Important Information about Our Organizations and Physician Affiliation

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