Gene therapy successfully restores immune function in children with ADA-SCID
An experimental gene therapy has restored and maintained immune function in 59 of 62 children with ADA-SCID. Conducted by researchers from UCLA and Great Ormond Street Hospital, the study represents the largest follow-up to date, showing promising long-term outcomes.
An experimental gene therapy developed by researchers at UCLA, University College London, and Great Ormond Street Hospital has shown notable success in restoring and maintaining immune system function in 59 of 62 children diagnosed with adenosine deaminase-deficient severe combined immunodeficiency (ADA-SCID). This rare genetic disorder severely compromises the immune system, leaving affected children vulnerable to life-threatening infections. The therapy's effectiveness was highlighted in a study published in the New England Journal of Medicine, which reported long-term outcomes from treatments administered between 2012 and 2019. The results demonstrate a significant advancement in managing this previously untreatable condition, with 95% of the treated children showing restored immune function and a stable health profile.
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