Epigenetic Therapy to Treat Pitt-Hopkins Syndrome (PTHS)

Awardee: Andrew Kennedy

Institution: Bates College

Award Amount: $49,263

Funding Period: January 1, 2018 - December 31, 2018

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Self-immolating NanoSpermine (nSpm) as a novel treatment approach to restore normal spermine/spermidine ratios in Snyder-Robinson Syndrome (SRS) models.

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AAVrh.10-mediated Npc2 Gene transfer for the treatment of Npc2 Deficient Niemann-Pick disease