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

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

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

Stem Cell Reports ; 18(6): 1388, 2023 Jun 13.

Article in En | MEDLINE | ID: mdl-37315526

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https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10277829
https://dx.doi.org/10.1016/j.stemcr.2023.05.008

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Full text: 1 Collection: 01-internacional Database: MEDLINE Language: En Journal: Stem Cell Reports Year: 2023 Document type: Article

Fulltext

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10277829
https://dx.doi.org/10.1016/j.stemcr.2023.05.008

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Full text: 1 Collection: 01-internacional Database: MEDLINE Language: En Journal: Stem Cell Reports Year: 2023 Document type: Article