[ExI] How One Round of Gene Therapy Fixed 48 Kids’ Immune Systems

[John Grigg]

"Gene therapy has shown promise in recent years for treating a range of
diseases, including sickle-cell anemia
<https://singularityhub.com/2019/12/17/gene-therapy-for-sickle-cell-anemia-looks-promising-but-its-riddled-with-controversy/>,
hemophilia
<https://www.sciencedirect.com/science/article/abs/pii/S1473050219301570>,
various forms of inherited blindness
<https://singularityhub.com/2020/03/03/gene-therapy-is-successfully-treating-a-common-form-of-inherited-blindness>,
mesothelioma <https://www.mesothelioma.com/treatment/gene-therapy/>,
and Duchenne
muscular dystrophy
<https://singularityhub.com/2020/07/30/a-year-after-getting-gene-therapy-boys-with-muscular-dystrophy-are-healthier-and-stronger/>.
A new success story may soon be added to this list, with the publication
yesterday of the outcomes of a clinical trial that used gene therapy to
cure a rare immune system disorder in infants.

The study, described in the *New England Journal of Medicine
<https://www.nejm.org/doi/full/10.1056/NEJMoa2027675>,* was carried out by
researchers from UCLA and Great Ormond Street Hospital in London over the
course of five years, beginning in 2012."

https://singularityhub.com/2021/05/13/how-one-round-of-gene-therapy-repaired-a-dysfunction-in-48-kids-immune-systems/
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