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

• Main Authors: Zixiang Yan (Author), Yuqin Yao (Author), Luyao Li (Author), Lingqiong Cai (Author), Haiwei Zhang (Author), Shenghai Zhang (Author), Qingquan Xiao (Author), Xing Wang (Author), Erwei Zuo (Author), Chunlong Xu (Author), Jihong Wu (Author), Hui Yang (Author)
• Format: Book
• Published: Elsevier, 2023-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...