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Breaking Barriers: Pioneering of Artemis-SCID Gene Therapy

Artemis-SCID is a severe form of immunodeficiency caused by mutations in a specific gene. A simple infection can easily kill children with Artemis-SCID in their first year of life if it is left untreated. Even with a bone marrow transplant – the current standard treatment – children with SCID often fail to develop a normal immune system and require repeated infusions to stay alive. UCSF Pediatrics professors Morton Cowan, MD, and Jennifer Puck, MD, are leading a clinical trial that involves transferring a normal copy of the mutated gene into patients’ own stem cells. The study aims to determine whether the procedure is safe, feasible and results in a normal immune system up to 15 years later. For more info: https://clinicaltrials.ucsf.edu/trial...