Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants
Severe Combined Immunodeficiency Disease, X-linkedSCIDSCID-X1X-linked SCIDimmunodeficiencygene therapyImmunologic Deficiency SyndromesSevere Combined ImmunodeficiencyX-Linked Combined Immunodeficiency DiseasesBusulfanCL20-i4-EF1α-hγc-OPTCliniMacs
Lead Scientist at UCSF
- Mort Cowan
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.