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

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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 pl...

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Detalles Bibliográficos
Autores principales: Boon, Nanda, Lu, Xuefei, Andriessen, Charlotte A., Moustakas, Ioannis, Buck, Thilo M., Freund, Christian, Arendzen, Christiaan H., Böhringer, Stefan, Mei, Hailiang, Wijnholds, Jan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10202653/
https://www.ncbi.nlm.nih.gov/pubmed/37084726
http://dx.doi.org/10.1016/j.stemcr.2023.03.014

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10202653/
https://www.ncbi.nlm.nih.gov/pubmed/37084726
http://dx.doi.org/10.1016/j.stemcr.2023.03.014

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