Engineering blood stem cells for autologous transplants for lysosomal diseases: Correction of mucopolysaccharidosis type I using genome-edited hematopoietic stem and progenitor cells Gomez-Ospina, N., Scharenberg, S. G., Mantri, S., Nicolas, C., Bak, R. O., Porteus, M. H. ACADEMIC PRESS INC ELSEVIER SCIENCE. 2018: S54–S55

View details for DOI 10.1016/j.ymgme.2017.12.129

View details for Web of Science ID 000424963800122