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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...
| Autores principales: | , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Frontiers Media S.A.
2022
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| Materias: | |
| 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 |