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

MDBRCRB1

Feb 1

Written By Million Dollar Bike Ride

Awardee: Jan Winjholds

Institution: Leiden University Medical Center (LUMC)

Award Amount: $50,312

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

MDBRCRB12018

Million Dollar Bike Ride

Targeting phagosome maturation to restore dysfunctional retinal pigmented epithelium in CHM

CRB1-null human retinal organoids for disease modeling and gene therapy testing