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Gene replacement therapy restores RCBTB1 expression and cilium length in patient‐derived retinal pigment epithelium

Biallelic mutations in the RCBTB1 gene cause retinal dystrophy. Here, we characterized the effects of RCBTB1 gene deficiency in retinal pigment epithelial (RPE) cells derived from a patient with RCBTB1‐associated retinopathy and restored RCBTB1 expression in these cells using adeno‐associated viral...

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Detalles Bibliográficos
Autores principales:	Huang, Zhiqin, Zhang, Dan, Chen, Shang‐Chih, Jennings, Luke, Carvalho, Livia S., Fletcher, Sue, Chen, Fred K., McLenachan, Samuel
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2021
Materias:	Original Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8572767/ https://www.ncbi.nlm.nih.gov/pubmed/34617687 http://dx.doi.org/10.1111/jcmm.16911

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