ASGCT 2021: The Gene Therapy Show Must Go On genengnews.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from genengnews.com Daily Mail and Mail on Sunday newspapers.
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A novel gene therapy for severe combined immunodeficiency from adenosine deaminase deficiency (ADA-SCID) restored immune cell counts without death or need for enzyme replacement therapy, researchers reported.
Survival without needing to re-initiate pegylated ADA enzyme replacement therapy, or have a rescue allogeneic hematopoietic stem cell transplantation (AHCT), was 95% to 97% at last follow-up at 24 or 36 months across three studies pooled together by Donald B. Kohn, MD, of the University of California Los Angeles, and colleagues.
Those rates compare favorably with the current standard of care, AHCT, for which overall survival of 65% to 88% and event-free survival of 56% has been reported, they stated in the