Article
Knockout and Replacement Gene Surgery to Treat Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa.
Human gene therapy - 1 Mar 2024
Sun Xuehan, Liang Chen, Chen Yangcan, Cui Tongtong, Han Jiabao, Dai Moyu, Zhang Ying, Zhou Qi, Li Wei
Abstract excerpt
Mutations in the rhodopsin (RHO) gene are the predominant causes of autosomal dominant retinitis pigmentosa (adRP). Given the diverse gain-of-function mutations, therapeutic strategies targeting specific sequences face significant challenges. Here, we provide a universal approach to conquer this problem: we have devised a CRISPR-Cas12i-based, mutation-independent gene knockout and replacement compound therapy...
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