AAVrh.10-mediated Npc2 Gene transfer for the treatment of Npc2 Deficient Niemann-Pick disease
Awardee: Dolan Sondhi
Institution: Weill Cornell Medicine
Award Amount: $49,645
Funding Period: January 1, 2018 - December 31, 2018
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Epigenetic Therapy to Treat Pitt-Hopkins Syndrome (PTHS)
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