Allele-specific gene-editing approach for vision loss restoration in RHO-associated retinitis pigmentosa

Mutant RHO is the most frequent genetic cause of autosomal dominant retinitis pigmentosa (adRP). Here, we developed an allele-specific gene-editing therapeutic drug to selectively target the human T17M RHO mutant allele while leaving the wild-type RHO allele intact for the first time. We identified...

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Detalles Bibliográficos
Autores principales: Liu, Xiaozhen, Qiao, Jing, Jia, Ruixuan, Zhang, Fan, Meng, Xiang, Li, Yang, Yang, Liping
Formato: Online Artículo Texto
Lenguaje:English
Publicado: eLife Sciences Publications, Ltd 2023
Materias:
Genetics and Genomics
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10279453/
https://www.ncbi.nlm.nih.gov/pubmed/37272616
http://dx.doi.org/10.7554/eLife.84065

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10279453/
https://www.ncbi.nlm.nih.gov/pubmed/37272616
http://dx.doi.org/10.7554/eLife.84065

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