UC San Francisco (UCSF) | Breaking Barriers: Pioneering of Artemis-SCID Gene Therapy @ucsf | Uploaded 1 year ago | Updated 2 minutes ago
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/NCT03538899
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/NCT03538899
