CIRM Funded Clinical Trials

Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency Using a Lentiviral Vector to Transduce Autologous CD34 Hematopoietic Stem Cells

Disease Area:
Severe Combined Immunodeficiency, Artemis deficient (ART-SCID)
CIRM Grant:
CLIN2-10830 (Active)
Award Value:
Trial Sponsor:
University of California, San Francisco
Trial Stage:
Phase 1
Trial Status:
Targeted Enrollment:
25 ID:


UC San Francisco researchers aim to reĀ­pair the damaged immune system of children born with severe combined immunodeficiency (SCID), a genetic blood disorder in which even a mild infection can be fatal. This trial will focus on SCID patients who have mutations in a gene called Artemis, the most difficult form of SCID to treat when using a standard bone marrow transplant from a healthy donor. The team will genetically modify the patient’s own blood stem cells with a functional copy of Artemis, with the goal of creating a new blood system and restoring the health of the immune system.


Open label, single arm study.


Safety and efficacy. Multilineage engraftment persistence and B cell reconstitution.

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