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

Boon N, Lu X, Andriessen CA, Moustakas I, Buck TM, Freund C, Arendzen CH, Bohringer S, Mei H, Wijnholds J
Stem Cell Reports. 2023 May 9;18(5):1123-1137. doi: 10.1016/j.stemcr.2023.03.014. Epub 2023 Apr 20. (Link opens in a new window) PubMed (Link opens in a new window) Article

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