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

Awardee: Jan Winjholds

Institution: Leiden University Medical Center (LUMC)

Award Amount: $50,312

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

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Targeting phagosome maturation to restore dysfunctional retinal pigmented epithelium in CHM

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