Toward CRISPR Therapies for Cardiomyopathies.
作者信息
Nishiyama Takahiko, Bassel-Duby Rhonda, Olson Eric N
机构信息
Department of Molecular Biology, Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Center, and Hamon Center for Regenerative Science and Medicine, University of Texas Southwestern Medical Center, Dallas, TX.
出版信息
Circulation. 2021 Nov 9;144(19):1525-1527. doi: 10.1161/CIRCULATIONAHA.121.057203. Epub 2021 Nov 8.
Abstract
摘要
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In vivo base editing rescues Hutchinson-Gilford progeria syndrome in mice.体内碱基编辑拯救亨廷顿病样核纤层蛋白病小鼠。
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Myoediting: Toward Prevention of Muscular Dystrophy by Therapeutic Genome Editing.肌编辑：通过治疗性基因组编辑预防肌肉疾病。
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