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Treatment of autosomal dominant retinitis pigmentosa caused by RHO-P23H mutation with high-fidelity Cas13X in mice

Mutations in Rhodopsin (RHO) gene commonly cause autosomal dominant retinitis pigmentosa (adRP) without effective therapeutic treatment so far. Compared with genomic DNA-targeting CRISPR-Cas9 system, Cas13 edits RNA for therapeutic applications, avoiding the risk of causing permanent changes in the...

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Detalles Bibliográficos
Autores principales:	Yan, Zixiang, Yao, Yuqin, Li, Luyao, Cai, Lingqiong, Zhang, Haiwei, Zhang, Shenghai, Xiao, Qingquan, Wang, Xing, Zuo, Erwei, Xu, Chunlong, Wu, Jihong, Yang, Hui
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2023
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10445100/ https://www.ncbi.nlm.nih.gov/pubmed/37621413 http://dx.doi.org/10.1016/j.omtn.2023.08.002

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