Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency Using a Lentiviral Vector to Transduce Autologous CD34 Hematopoietic Stem Cells
UC San Francisco researchers aim to repair 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.