AAV-Mediated Gene Augmentation Therapy of CRB1 Patient-Derived Retinal Organoids Restores the Histological and Transcriptional Retinal Phenotype

Crumbs homolog 1 (CRB1) patient-derived PSC were differentiated into CRB1 retinal organoids that showed diminished expression of variant CRB1 protein observed by immunohistochemical analysis.