Gene therapy restores immune function in children with ADA-SCID

timesnownews.com

Experimental gene therapy has successfully restored immune function in children with the rare, life-threatening disorder ADA-SCID. The therapy uses a modified lentivirus to deliver a healthy ADA gene into a child's blood stem cells, with long-term follow-up showing stable immune function and no significant complications. This advancement, published in the New England Journal of Medicine, offers hope for wider accessibility and potential FDA approval within years, transforming treatment for children with this condition.


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