Article
CRISPR genome surgery in a novel humanized model for autosomal dominant retinitis pigmentosa.
Molecular therapy : the journal of the American Society of Gene Therapy - 6 Apr 2022
Wu Wen-Hsuan, Tsai Yi-Ting, Huang I-Wen, Cheng Chia-Hua, Hsu Chun-Wei, Cui Xuan, Ryu Joseph, Quinn Peter M J, Caruso Salvatore Marco, Lin Chyuang-Sheng, Tsang Stephen H
Abstract excerpt
Mutations in rhodopsin (RHO) are the most common causes of autosomal dominant retinitis pigmentosa (adRP), accounting for 20% to 30% of all cases worldwide. However, the high degree of genetic heterogeneity makes development of effective therapies cumbersome. To provide a universal solution to RHO-related adRP, we devised a CRISPR-based, mutation-independent gene ablation and replacement (AR) compound therapy...
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