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Article

AAV-mediated gene augmentation therapy of CRB1 patient-derived retinal organoids restores the histological and transcriptional retinal phenotype.

Stem cell reports - 9 May 2023

Boon Nanda, Lu Xuefei, Andriessen Charlotte A, Moustakas Ioannis, Buck Thilo M, Freund Christian, Arendzen Christiaan H, Böhringer Stefan, Mei Hailiang, Wijnholds Jan

Abstract excerpt

Retinitis pigmentosa and Leber congenital amaurosis are inherited retinal dystrophies that can be caused by mutations in the Crumbs homolog 1 (CRB1) gene. CRB1 is required for organizing apical-basal polarity and adhesion between photoreceptors and Müller glial cells. CRB1 patient-derived induced pluripotent stem cells were differentiated into CRB1 retinal organoids that showed diminished expression of variant...

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Identifiers

PMID: 37084726
DOI: 10.1016/j.stemcr.2023.03.014
PMCID: PMC10202653

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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