Micro-dystrophin Genes Bring Hope of an Effective Therapy for Duchenne Muscular Dystrophy.
作者信息
Davies Kay E, Guiraud Simon
机构信息
MDUK Oxford Neuromuscular Centre, Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford OX1 3PT, UK.
MDUK Oxford Neuromuscular Centre, Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford OX1 3PT, UK.
出版信息
Mol Ther. 2019 Mar 6;27(3):486-488. doi: 10.1016/j.ymthe.2019.01.019. Epub 2019 Feb 12.
Abstract
摘要
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本文引用的文献
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Development of Novel Micro-dystrophins with Enhanced Functionality.新型功能增强型微肌营养不良蛋白的研发。
Mol Ther. 2019 Mar 6;27(3):623-635. doi: 10.1016/j.ymthe.2019.01.002. Epub 2019 Feb 1.
2
Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy.系统性 AAV 微肌营养不良蛋白基因治疗杜氏肌营养不良症。
Mol Ther. 2018 Oct 3;26(10):2337-2356. doi: 10.1016/j.ymthe.2018.07.011. Epub 2018 Jul 17.
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Dystrophin's central domain forms a complex filament that becomes disorganized by in-frame deletions.抗肌萎缩蛋白的中央结构域形成一个复杂的细丝，该细丝通过框内缺失而变得紊乱。
J Biol Chem. 2018 May 4;293(18):6637-6646. doi: 10.1074/jbc.M117.809798. Epub 2018 Mar 13.
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Cardiac-Specific Expression of ΔH2-R15 Mini-Dystrophin Normalized All Electrocardiogram Abnormalities and the End-Diastolic Volume in a 23-Month-Old Mouse Model of Duchenne Dilated Cardiomyopathy.心脏特异性表达ΔH2-R15 微肌营养不良蛋白使 23 月龄 Duchenne 扩张型心肌病小鼠模型的所有心电图异常和舒张末期容积正常化。
Hum Gene Ther. 2018 Jul;29(7):737-748. doi: 10.1089/hum.2017.144. Epub 2018 Mar 22.
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Nat Commun. 2017 Jul 25;8:16105. doi: 10.1038/ncomms16105.
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Progress toward Gene Therapy for Duchenne Muscular Dystrophy.杜氏肌营养不良症基因治疗的进展
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The Pathogenesis and Therapy of Muscular Dystrophies.肌肉萎缩症的发病机制与治疗
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J Clin Invest. 2009 Mar;119(3):624-35. doi: 10.1172/JCI36612. Epub 2009 Feb 23.
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