A CEP290 C-Terminal Domain Complements the Mutant CEP290 of Rd16 Mice In Trans and Rescues Retinal Degeneration

Mutations in CEP290 cause ciliogenesis defects, leading to diverse clinical phenotypes, including Leber congenital amaurosis (LCA). Gene therapy for CEP290-associated diseases is hindered by the 7.4 kb CEP290 coding sequence, which is difficult to deliver in vivo. The multi-domain structure of the C...

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Detalhes bibliográficos
Publicado no:Cell Rep
Main Authors: Mookherjee, Suddhasil, Chen, Holly Yu, Isgrig, Kevin, Yu, Wenhan, Hiriyanna, Suja, Levron, Rivka, Li, Tiansen, Colosi, Peter, Chien, Wade, Swaroop, Anand, Wu, Zhijian
Formato: Artigo
Idioma:Inglês
Publicado em: 2018
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6245950/
https://ncbi.nlm.nih.gov/pubmed/30332642
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.celrep.2018.09.043
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