Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants
Severe Combined Immunodeficiency Disease, X-linked SCID SCID-X1 X-linked SCID immunodeficiency gene therapy Immunologic Deficiency Syndromes Severe Combined Immunodeficiency X-Linked Combined Immunodeficiency Diseases Busulfan CL20-i4-EF1α-hγc-OPT CliniMacs
Lead Scientist at UCSF
- Mort Cowan, MD
California Institute of Regenerative Medicine vGene Correction of Autologous Hematopoietic Stem Cells in Artemis Deficient SCID Goal is to complete the preclinical testing necessary for a clinical gene therapy trial in children with Artemis-SCID.