CRISPR/Cas9 Flexes Its Muscles: In Vivo Somatic Gene Editing for Muscular Dystrophy.
作者信息
VandenDriessche Thierry, Chuah Marinee K
机构信息
Department of Gene Therapy and Regenerative Medicine, Free University of Brussels (VUB), Brussels, Belgium.
Center for Molecular and Vascular Biology, Department of Cardiovascular Sciences, University of Leuven, Leuven, Belgium.
出版信息
Mol Ther. 2016 Mar;24(3):414-6. doi: 10.1038/mt.2016.29.
Abstract
摘要
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Mol Ther Nucleic Acids. 2016 Jan 26;5(1):e283. doi: 10.1038/mtna.2015.58.
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Selection-free gene repair after adenoviral vector transduction of designer nucleases: rescue of dystrophin synthesis in DMD muscle cell populations.腺病毒载体转导设计核酸酶后无需筛选的基因修复：在杜氏肌营养不良症（DMD）肌肉细胞群体中恢复肌营养不良蛋白的合成
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In vivo gene editing in dystrophic mouse muscle and muscle stem cells.营养不良小鼠肌肉和肌肉干细胞中的体内基因编辑。
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