Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model
Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model
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DOI：
10.1073/pnas.240335297
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发表时间：
2000-12-05
影响因子：
11.1
通讯作者：
Xiao, X
中科院分区：
文献类型：
--
作者：
Wang, B;Li, J;Xiao, X
杜氏肌营养不良症（DMD）是最常见和致命的遗传性肌肉疾病，由肌营养不良蛋白基因的隐性突变引起。每3500名配偶中就有一人患有DMD，但目前还没有治疗方法。主要使用成肌细胞移植和腺病毒介导的基因转移的基因治疗方法，已经取得了有限的成功。腺相关病毒（AAV）载体虽然被证明对于肌肉基因转移是上级的，但太小（5 kb）而不能包装14-kb肌营养不良蛋白cDNA。在这里，我们创造了一系列的小肌萎缩蛋白基因（
Duchenne muscular dystrophy (DMD) is the most common and lethal genetic muscle disorder, caused by recessive mutations in the dystrophin gene. One of every 3.500 mates suffers from DMD, yet no treatment is currently available. Genetic therapeutic approaches, using primarily myoblast transplantation and adenovirus-mediated gene transfer, have met with limited success. Adenoassociated virus (AAV) vectors, although proven superior for muscle gene transfer, are too small (5 kb) to package the 14-kb dystrophin cDNA. Here we have created a series of minidystrophin genes (