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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Main Authors: Ying Hsu, Sajag Bhattarai, Jacob M. Thompson, Angela Mahoney, Jacintha Thomas, Sara K. Mayer, Poppy Datta, Janelle Garrison, Charles C. Searby, Luk H. Vandenberghe, Seongjin Seo, Val C. Sheffield, Arlene V. Drack
Format: Article
Language:English
Published: Elsevier 2023-03-01
Series:Molecular Therapy: Nucleic Acids
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Online Access:http://www.sciencedirect.com/science/article/pii/S2162253122003286