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The Harvard Gazette: New drug target for Rett syndrome

February 2, 2016

Researchers identify potential new route to therapy

Harvard Stem Cell Institute (HSCI) researchers have identified a faulty signaling pathway that, when corrected in mice, ameliorates the symptoms of Rett syndrome, a devastating neurological condition. The findings could lead to the discovery of compounds or drugs that may benefit children affected by the disease, says neurobiologist Jeffrey Macklis, a member of the HSCI Executive Committee.

The research was published recently in Nature Communications. Noriyuki Kishi and Jessica MacDonald...

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