美国《科学》杂志日前发表的3项独立研究显示,被誉为“基因剪刀”的CRISPR基因编辑技术有望用来治疗一种肌肉萎缩遗传病——杜兴肌营养不良症。美国杜克大学、得克萨斯大学和哈佛大学的研究人员分别通过编辑一个涉及肌肉功能的基因,成功让罹患杜兴肌营养不良症的小鼠恢复一定程度的肌肉功能。这也是研究人员首次成功利用基因编辑技术给活体哺乳动物治疗遗传疾病。杜兴肌营养不良症由抗 Three independent studies published recently by the U.S. “Science” magazine show that the CRISPR gene editing technology known as “Gene Scissors ” is expected to be used to treat Duchenine muscular dystrophy. Researchers at Duke University, the University of Texas, and Harvard University have successfully restored some muscle function in mice with Duchenne muscular dystrophy by editing a gene involved in muscle function, respectively. It is also the first time that researchers have successfully used gene editing techniques to treat genetic diseases in living mammals. Duchenne muscular dystrophy by the anti