Pitt-Hopkins syndrome | sciencenewsnet.in

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Gene Therapy Could Treat Pitt-Hopkins Syndrome, Proof-of-Concept Study Suggests

May 10, 2022 sarah JonasResearch Results

University of North Carolina School of Medicine scientists, who report their results in the journal eLife, devised an experimental, gene-therapy-like technique to restore the normal activity of the gene deficient in people with Pitt-Hopkins syndrome.