Gene Replacement Therapy for APBD

Awardee: Berge Minassian

Institution: UT Southwestern Medical Center

Award Amount: $53,455

Funding Period: February 1, 2019 - January 31, 2020

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Pilot study to develop a small molecule activator of TCF4 as a treatment for Pitt-Hopkins syndrome

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Targeting phagosome maturation to restore dysfunctional retinal pigmented epithelium in CHM