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

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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.
[Stem Cell Reports]
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