Targeting phagosome maturation to restore dysfunctional retinal pigmented epithelium in CHM

MDBRCHM

Feb 1

Written By Million Dollar Bike Ride

Awardee: Kathleen Boesze-Battaglia

Institution: University of Pennsylvania

Award Amount: $101,740

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

MDBRCHM2018

Million Dollar Bike Ride

Gene Replacement Therapy for APBD

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