Matthew Porteus

Associate Professor of Pediatrics (Cancer Biology)
Dr. Porteus was raised in California and was a local graduate of Gunn High School before completing A.B. degree in “History and Science” at Harvard University where he graduated Magna Cum Laude and wrote an thesis entitled “Safe or Dangerous Chimeras: The recombinant DNA controversy as a conflict between differing socially constructed interpretations of recombinant DNA technology.” He then returned to the area and completed his combined MD, PhD at Stanford Medical School with his PhD focused on understanding the molecular basis of mammalian forebrain development with his PhD thesis entitled “Isolation and Characterization of TES-1/DLX-2: A Novel Homeobox Gene Expressed During Mammalian Forebrain Development.” After completion of his dual degree program, he was an intern and resident in Pediatrics at Boston Children’s Hospital and then completed his Pediatric Hematology/Oncology fellowship in the combined Boston Chidlren’s Hospital/Dana Farber Cancer Institute program. For his fellowship and post-doctoral research he worked with Dr. David Baltimore at MIT and CalTech where he began his studies in developing homologous recombination as a strategy to correct disease causing mutations in stem cells as definitive and curative therapy for children with genetic diseases of the blood, particularly sickle cell disease. Following his training with Dr. Baltimore, he took an independent faculty position at UT Southwestern in the Departments of Pediatrics and Biochemistry before again returning to Stanford in 2010 as an Associate Professor. During this time his work has been the first to demonstrate that gene correction could be achieved in human cells at frequencies that were high enough to potentially cure patients and is considered one of the pioneers and founders of the field of genome editing—a field that now encompasses thousands of labs and several new companies throughout the world. His research program continues to focus on developing genome editing by homologous recombination as curative therapy for children with genetic diseases but also has interests in the clonal dynamics of heterogeneous populations and the use of genome editing to better understand diseases that affect children including infant leukemias and genetic diseases that affect the muscle. Clinically, Dr. Porteus attends at the Lucille Packard Children’s Hospital where he takes care of pediatric patients undergoing hematopoietic stem cell transplantation.

Professional Education

Fellowship: Children's Hospital Boston (1999) MA

Residency: Children's Hospital Boston (1996) MA

Medical Education: Stanford University School of Medicine (1994) CA

Board Certification: Pediatric Hematology-Oncology, American Board of Pediatrics (2000)

Clinical Trials

Clinical trials are research studies that evaluate a new medical approach, device, drug, or other treatment. As a Stanford Health Care patient, you may have access to the latest, advanced clinical trials.

Open trials refer to studies currently accepting participants. Closed trials are not currently enrolling, but may open in the future.

Quantifying Genome-Editing Outcomes at Endogenous Loci with SMRT Sequencing
Hendel, A., Kildebeck, E. J., Fine, E. J., Clark, J. T., Punjya, N., & Porteus, M. H. (2014). Quantifying Genome-Editing Outcomes at Endogenous Loci with SMRT Sequencing. CELL REPORTS, 7(1), 293-305.

SAPTA: a new design tool for improving TALE nuclease activity
Lin, Y., Fine, E. J., Zheng, Z., Antico, C. J., Voit, R. A., & Bao, G. (2014). SAPTA: a new design tool for improving TALE nuclease activity. NUCLEIC ACIDS RESEARCH, 42(6).

Nuclease-mediated gene editing by homologous recombination of the human globin locus
Voit, R. A., Hendel, A., Pruett-Miller, S. M., & Porteus, M. H. (2014). Nuclease-mediated gene editing by homologous recombination of the human globin locus. NUCLEIC ACIDS RESEARCH, 42(2), 1365-1378.

Phosphorylation of EXO1 by CDKs 1 and 2 regulates DNA end resection and repair pathway choice.
Tomimatsu, N., Mukherjee, B., Catherine Hardebeck, M., Ilcheva, M., Vanessa Camacho, C., & Burma, S. (2014). Phosphorylation of EXO1 by CDKs 1 and 2 regulates DNA end resection and repair pathway choice. Nature communications, 5, 3561-?.

An Erythroid Enhancer of BCL11A Subject to Genetic Variation Determines Fetal Hemoglobin Level
Bauer, D. E., Kamran, S. C., Lessard, S., Xu, J., Fujiwara, Y., & Orkin, S. H. (2013). An Erythroid Enhancer of BCL11A Subject to Genetic Variation Determines Fetal Hemoglobin Level. SCIENCE, 342(6155), 253-257.

Receptor-mediated delivery of engineered nucleases for genome modification
Chen, Z., Jaafar, L., Agyekum, D. G., Xiao, H., Wade, M. F., & Meiler, S. E. (2013). Receptor-mediated delivery of engineered nucleases for genome modification. NUCLEIC ACIDS RESEARCH, 41(19).

Newborn screening for severe combined immunodeficiency and T-cell lymphopenia in California: Results of the first 2 years
Kwan, A., Church, J. A., Cowan, M. J., Agarwal, R., Kapoor, N., & Puck, J. M. (2013). Newborn screening for severe combined immunodeficiency and T-cell lymphopenia in California: Results of the first 2 years. JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY, 132(1), 140-U245.

Generation of an HIV Resistant T-cell Line by Targeted "Stacking" of Restriction Factors
Voit, R. A., McMahon, M. A., Sawyer, S. L., & Porteus, M. H. (2013). Generation of an HIV Resistant T-cell Line by Targeted "Stacking" of Restriction Factors. MOLECULAR THERAPY, 21(4), 786-795.

Expanding the Repertoire of Target Sites for Zinc Finger Nuclease-mediated Genome Modification
Wilson, K. A., McEwen, A. E., Pruett-Miller, S. M., Zhang, J., Kildebeck, E. J., & Porteus, M. H. (2013). Expanding the Repertoire of Target Sites for Zinc Finger Nuclease-mediated Genome Modification. MOLECULAR THERAPY-NUCLEIC ACIDS, 2.

Design and Development of Artificial Zinc Finger Transcription Factors and Zinc Finger Nucleases to the hTERT Locus
Wilson, K. A., Chateau, M. L., & Porteus, M. H. (2013). Design and Development of Artificial Zinc Finger Transcription Factors and Zinc Finger Nucleases to the hTERT Locus. MOLECULAR THERAPY-NUCLEIC ACIDS, 2.

A Crisper Look at Genome Editing: RNA-guided Genome Modification
Damian, M., & Porteus, M. H. (2013). A Crisper Look at Genome Editing: RNA-guided Genome Modification. MOLECULAR THERAPY, 21(4), 719-721.

A crisper look at genome editing: RNA-guided genome modification.
Damian, M., & Porteus, M. H. (2013). A crisper look at genome editing: RNA-guided genome modification. Molecular therapy : the journal of the American Society of Gene Therapy, 21(4), 720-722.

A survey of ex vivo/in vitro transduction efficiency of mammalian primary cells and cell lines with Nine natural adeno-associated virus (AAV1-9) and one engineered adeno-associated virus serotype
Ellis, B. L., Hirsch, M. L., Barker, J. C., Connelly, J. P., Steininger, R. J., & Porteus, M. H. (2013). A survey of ex vivo/in vitro transduction efficiency of mammalian primary cells and cell lines with Nine natural adeno-associated virus (AAV1-9) and one engineered adeno-associated virus serotype. VIROLOGY JOURNAL, 10.

Zinc-finger nuclease-mediated gene correction using single AAV vector transduction and enhancement by Food and Drug Administration-approved drugs
Ellis, B. L., HIRSCH, M. L., Porter, S. N., Samulski, R. J., & Porteus, M. H. (2013). Zinc-finger nuclease-mediated gene correction using single AAV vector transduction and enhancement by Food and Drug Administration-approved drugs. GENE THERAPY, 20(1), 35-42.

Gene therapy for primary immunodeficiencies
Kildebeck, E., Checketts, J., & Porteus, M. (2012). Gene therapy for primary immunodeficiencies. CURRENT OPINION IN PEDIATRICS, 24(6), 731-738.

Engineering the immune system to cure genetic diseases, HIV, and cancer Editorial overview
Porteus, M. H., & Fischer, A. (2012). Engineering the immune system to cure genetic diseases, HIV, and cancer Editorial overview. CURRENT OPINION IN IMMUNOLOGY, 24(5), 576-579.

Development of nuclease-mediated site-specific genome modification.
Wirt, S. E., & Porteus, M. H. (2012). Development of nuclease-mediated site-specific genome modification. Current opinion in immunology, 24(5), 609-616.

Gene editing: not just for translation anymore
McMahon, M. A., Rahdar, M., & Porteus, M. (2012). Gene editing: not just for translation anymore. NATURE METHODS, 9(1), 28-31.

Viral Single-Strand DNA Induces p53-Dependent Apoptosis in Human Embryonic Stem Cells
Hirsch, M. L., Fagan, B. M., Dumitru, R., Bower, J. J., Yadav, S., & Samulski, R. J. (2011). Viral Single-Strand DNA Induces p53-Dependent Apoptosis in Human Embryonic Stem Cells. PLOS ONE, 6(11).

Seeing the light: integrating genome engineering with double-strand break repair
Porteus, M. (2011). Seeing the light: integrating genome engineering with double-strand break repair. NATURE METHODS, 8(8), 628-630.

Translating the Lessons From Gene Therapy to the Development of Regenerative Medicine
Porteus, M. (2011). Translating the Lessons From Gene Therapy to the Development of Regenerative Medicine. MOLECULAR THERAPY, 19(3), 439-441.

Homologous recombination-based gene therapy for the primary immunodeficiencies
Porteus, M. (2011). Homologous recombination-based gene therapy for the primary immunodeficiencies. YEAR IN HUMAN AND MEDICAL GENETICS: INBORN ERRORS OF IMMUNITY II, 1246, 131-140.

COCCIDIOIDAL ANTIGEN-REACTIVE CD4(+) T-LYMPHOCYTES IN THE CEREBROSPINAL-FLUID IN COCCIDIOIDES-IMMITIS MENINGITIS
Vollmer, T. L., Gaiser, C., DELLOCA, R. L., Porteus, M., Steinman, L., & Stevens, D. A. (1995). COCCIDIOIDAL ANTIGEN-REACTIVE CD4(+) T-LYMPHOCYTES IN THE CEREBROSPINAL-FLUID IN COCCIDIOIDES-IMMITIS MENINGITIS. JOURNAL OF MEDICAL AND VETERINARY MYCOLOGY, 33(1), 43-48.

DLX-P, MASH-1, AND MAP-5 EXPRESSION AND BROMODEOXYURIDINE INCORPORATION DEFINE MOLECULARLY DISTINCT CELL-POPULATIONS IN THE EMBRYONIC MOUSE FOREBRAIN
Porteus, M. H., Bulfone, A., Liu, J. K., Puelles, L., Lo, L. C., & Rubenstein, J. Lr. (1994). DLX-P, MASH-1, AND MAP-5 EXPRESSION AND BROMODEOXYURIDINE INCORPORATION DEFINE MOLECULARLY DISTINCT CELL-POPULATIONS IN THE EMBRYONIC MOUSE FOREBRAIN. JOURNAL OF NEUROSCIENCE, 14(11), 6370-6383.

DLX2 (TES1), A HOMEOBOX GENE OF THE DISTAL-LESS FAMILY, ASSIGNED TO CONSERVED REGIONS ON HUMAN AND MOUSE CHROMOSOMES-2
Ozcelik, T., Porteus, M. H., Rubenstein, J. Lr., & FRANCKE, U. (1992). DLX2 (TES1), A HOMEOBOX GENE OF THE DISTAL-LESS FAMILY, ASSIGNED TO CONSERVED REGIONS ON HUMAN AND MOUSE CHROMOSOMES-2. GENOMICS, 13(4), 1157-1161.

ISOLATION AND CHARACTERIZATION OF A LIBRARY OF CDNA CLONES THAT ARE PREFERENTIALLY EXPRESSED IN THE EMBRYONIC TELENCEPHALON
Porteus, M. H., BRICE, A. Ej., Bulfone, A., Usdin, T. B., CIARANELLO, R. D., & Rubenstein, J. Lr. (1992). ISOLATION AND CHARACTERIZATION OF A LIBRARY OF CDNA CLONES THAT ARE PREFERENTIALLY EXPRESSED IN THE EMBRYONIC TELENCEPHALON. MOLECULAR BRAIN RESEARCH, 12(1-3), 7-22.

ISOLATION AND CHARACTERIZATION OF A NOVEL CDNA CLONE ENCODING A HOMEODOMAIN THAT IS DEVELOPMENTALLY REGULATED IN THE VENTRAL FOREBRAIN
Porteus, M. H., Bulfone, A., CIARANELLO, R. D., & Rubenstein, J. Lr. (1991). ISOLATION AND CHARACTERIZATION OF A NOVEL CDNA CLONE ENCODING A HOMEODOMAIN THAT IS DEVELOPMENTALLY REGULATED IN THE VENTRAL FOREBRAIN. NEURON, 7(2), 221-229.

SUBTRACTIVE HYBRIDIZATION SYSTEM USING SINGLE-STRANDED PHAGEMIDS WITH DIRECTIONAL INSERTS
Rubenstein, J. Lr., BRICE, A. Ej., CIARANELLO, R. D., Denney, D., Porteus, M. H., & Usdin, T. B. (1990). SUBTRACTIVE HYBRIDIZATION SYSTEM USING SINGLE-STRANDED PHAGEMIDS WITH DIRECTIONAL INSERTS. NUCLEIC ACIDS RESEARCH, 18(16), 4833-4842.

VALIDATION OF A MODEL OF NON-RHEGMATOGENOUS RETINAL-DETACHMENT
Marmor, M. F., Porteus, M., Negi, A., & Immel, J. (1984). VALIDATION OF A MODEL OF NON-RHEGMATOGENOUS RETINAL-DETACHMENT. CURRENT EYE RESEARCH, 3(3), 515-518.