A First Step in Viral Gene Therapy for Muscular Dystrophy.
作者信息
Hirano Michio
机构信息
Columbia University Medical Center, 630 West 168th Street, P&S 4-423, New York, NY 10032, USA.
出版信息
Curr Neurol Neurosci Rep. 2010 Mar;10(2):71-72. doi: 10.1007/s11910-010-0090-x.
Abstract
摘要
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Gene therapy for muscular dystrophy reaches human clinical trial.
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A single intravenous injection of adeno-associated virus serotype-9 leads to whole body skeletal muscle transduction in dogs.单次静脉注射9型腺相关病毒可导致犬全身骨骼肌转导。
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Limb-girdle muscular dystrophies--from genetics to molecular pathology.肢带型肌营养不良症——从遗传学到分子病理学
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Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning.通过干细胞基因治疗联合非清髓性预处理纠正腺苷脱氨酶严重联合免疫缺陷症
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Dystrophin: the protein product of the Duchenne muscular dystrophy locus.肌营养不良蛋白：杜氏肌营养不良基因座的蛋白质产物。
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