Gene Therapy Restores Immune Function in Children with ADA-SCID
A gene therapy developed by UCLA and others has restored immune function in 59 out of 62 children with ADA-SCID, a severe genetic disorder, showing promising long-term results.
Looking ahead, the research team is preparing to apply for FDA approval for this gene therapy, aiming to accomplish this within the next two to three years. Collaborating with Rarity PBC, the team seeks to produce the therapy under pharmaceutical-grade conditions, which will help make it accessible to a wider population. The continuous advancements in this field mark an important step in treating genetic disorders and enhancing the quality of life for children affected by these conditions.
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