Tidball James G, Bertoni Carmen
Molecular, Cellular and Integrative Physiology Program, University of California, Los Angeles, Los Angeles, CA 90095-7239, USA; Department of Pathology and Laboratory Medicine, David Geffen School of Medicine at UCLA, University of California, Los Angeles, Los Angeles, CA 90095-1732, USA; Department of Integrative Biology and Physiology, University of California, Los Angeles, Los Angeles, CA 90095-7239, USA.
Department of Neurology, David Geffen School of Medicine at UCLA, University of California, Los Angeles, Los Angeles, CA 90095-1769, USA.
Cell Metab. 2014 Dec 2;20(6):927-9. doi: 10.1016/j.cmet.2014.11.011.
Myriad strategies have been explored to compensate for the lack of dystrophin or to skip mutations that cause the lethal disease Duchenne muscular dystrophy (DMD). A new study shows that gene editing strategies used by bacteria can be applied in zygotes of a mouse model of DMD to correct the genetic defect that causes muscular dystrophy (Long et al., 2014).
人们已经探索了无数策略来弥补肌营养不良蛋白的缺失，或跳过导致致命疾病杜氏肌营养不良症（DMD）的突变。一项新研究表明，细菌使用的基因编辑策略可应用于DMD小鼠模型的受精卵，以纠正导致肌肉营养不良的基因缺陷（Long等人，2014年）。
