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

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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Bibliographic Details
Main Authors: Zixiang Yan, Yuqin Yao, Luyao Li, Lingqiong Cai, Haiwei Zhang, Shenghai Zhang, Qingquan Xiao, Xing Wang, Erwei Zuo, Chunlong Xu, Jihong Wu, Hui Yang
Format: Article
Language:English
Published: Elsevier 2023-09-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: RNA/DNA editing
hfCas13X
rhodopsin
P23H mutation
retinitis pigmentosa
adRP
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253123002123

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http://www.sciencedirect.com/science/article/pii/S2162253123002123

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