Subretinal gene therapy delays vision loss in a Bardet-Biedl Syndrome type 10 mouse model

Blindness in Bardet-Biedl syndrome (BBS) is caused by dysfunction and loss of photoreceptor cells in the retina. BBS10, mutations of which account for approximately 21% of all BBS cases, encodes a chaperonin protein indispensable for the assembly of the BBSome, a cargo adaptor important for ciliary...

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Detalles Bibliográficos
Autores principales: Hsu, Ying, Bhattarai, Sajag, Thompson, Jacob M., Mahoney, Angela, Thomas, Jacintha, Mayer, Sara K., Datta, Poppy, Garrison, Janelle, Searby, Charles C., Vandenberghe, Luk H., Seo, Seongjin, Sheffield, Val C., Drack, Arlene V.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9841241/
https://www.ncbi.nlm.nih.gov/pubmed/36700052
http://dx.doi.org/10.1016/j.omtn.2022.12.007

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