Targeting phagosome maturation to restore dysfunctional retinal pigmented epithelium in CHM

Awardee: Kathleen Boesze-Battaglia

Institution: University of Pennsylvania

Award Amount: $101,740

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

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Gene Replacement Therapy for APBD

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AAV gene therapy on cultured human iPSC-derived retina with loss of CRB1 and CRB2