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

• Principais autores: Ying Hsu (Autor), Sajag Bhattarai (Autor), Jacob M. Thompson (Autor), Angela Mahoney (Autor), Jacintha Thomas (Autor), Sara K. Mayer (Autor), Poppy Datta (Autor), Janelle Garrison (Autor), Charles C. Searby (Autor), Luk H. Vandenberghe (Autor), Seongjin Seo (Autor), Val C. Sheffield (Autor), Arlene V. Drack (Autor)
• Formato: Livro
• Publicado em: Elsevier, 2023-03-01T00:00:00Z.
• Assuntos: article
• Acesso em linha: Connect to this object online.

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...