The ability to efficiently modify the genome using CRISPR technology has rapidly revolutionized biology and genetics and will soon transform medicine. Duchenne muscular dystrophy (DMD) represents one of the first monogenic disorders that has been investigated with respect to CRISPR-mediated correction of causal genetic mutations. DMD results from mutations in the gene encoding dystrophin, a scaffolding protein that maintains the integrity of striated muscles. Thousands of different dystrophin mutations have been identified in DMD patients, who suffer from a loss of ambulation followed by respiratory insufficiency, heart failure, and death by the third decade of life. Using CRISPR to bypass DMD mutations, dystrophin expression has been efficiently restored in human cells and mouse models of DMD. Here, we review recent progress toward the development of possible CRISPR therapies for DMD and highlight opportunities and potential obstacles in attaining this goal.

中文翻译：

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杜兴氏肌营养不良症的CRISPR校正。

使用CRISPR技术有效修饰基因组的能力迅速改变了生物学和遗传学，并将很快改变医学。Duchenne肌营养不良症（DMD）代表了最早的单基因疾病之一，该疾病已就CRISPR介导的因果遗传突变校正进行了研究。DMD是由编码抗肌萎缩蛋白（dystrophin）的基因突变产生的，肌营养蛋白是一种维持横纹肌完整性的支架蛋白。已在DMD患者中鉴定出成千上万种不同的肌营养不良蛋白突变，这些患者的走动能力下降，继而在生命的第三个十年中呼吸功能不全，心力衰竭和死亡。使用CRISPR绕过DMD突变，抗肌萎缩蛋白的表达已在人细胞和DMD小鼠模型中得到有效恢复。这里，