RNA-based therapies in animal models of Leber congenital amaurosis causing blindness

Leber congenital amaurosis (LCA) is a severe, genetically heterogeneous recessive eye disease in which ~ 35% of gene mutations are in-frame nonsense mutations coding for loss-of-function premature termination codons (PTCs) in mRNA. Nonsense suppression therapy allows read-through of PTCs leading to...

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Detalles Bibliográficos
Autores principales: Wang, Xia, Shan, Xianghong, Gregory-Evans, Kevin, Gregory-Evans, Cheryl Y
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2020
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8985810/
https://www.ncbi.nlm.nih.gov/pubmed/35692607
http://dx.doi.org/10.1093/pcmedi/pbaa009

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8985810/
https://www.ncbi.nlm.nih.gov/pubmed/35692607
http://dx.doi.org/10.1093/pcmedi/pbaa009

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