让基因编辑成为一种治疗现实。

Conboy Irina, Murthy Niren, Etienne Jessy, Robinson Zachery

Bioengineering, UC Berkeley, Berkeley, CA, 94720, USA.

F1000Res. 2018 Dec 21;7. doi: 10.12688/f1000research.16106.1. eCollection 2018.

This review discusses current bottlenecks in making CRISPR-Cas9-mediated genome editing a therapeutic reality and it outlines recent strategies that aim to overcome these hurdles as well as the scope of current clinical trials that pioneer the medical translation of CRISPR-Cas9. Additionally, this review outlines the specifics of disease-modifying gene editing in recessive versus dominant genetic diseases with the focus on genetic myopathies that are exemplified by Duchenne muscular dystrophy and myotonic dystrophies.

本综述讨论了将CRISPR-Cas9介导的基因组编辑转化为治疗现实的当前瓶颈，并概述了旨在克服这些障碍的最新策略，以及率先进行CRISPR-Cas9医学转化的当前临床试验的范围。此外，本综述概述了隐性与显性遗传病中疾病修饰基因编辑的具体情况，重点关注以杜氏肌营养不良症和强直性肌营养不良症为例的遗传性肌病。