Global spectrum of USH2A mutation in inherited retinal dystrophies: Prompt message for development of base editing therapy

PURPOSE: Mutation in the USH2A gene is the most common cause of inherited retinal dystrophy (IRD), including non-syndromic retinitis pigmentosa (RP) and Usher syndrome II (USH2). Gene editing and therapy targeting USH2A, especially the hotspot region, would benefit a large proportion of IRD patients...

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Detalles Bibliográficos
Autores principales: Su, Bing-Nan, Shen, Ren-Juan, Liu, Zhuo-Lin, Li, Yang, Jin, Zi-Bing
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Neuroscience
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9399374/
https://www.ncbi.nlm.nih.gov/pubmed/36034145
http://dx.doi.org/10.3389/fnagi.2022.948279

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9399374/
https://www.ncbi.nlm.nih.gov/pubmed/36034145
http://dx.doi.org/10.3389/fnagi.2022.948279

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