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ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF MICRO-DYSTROPHIN TO TREAT MUSCULAR DYSTROPHY.
ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF MICRO-DYSTROPHIN TO TREAT MUSCULAR DYSTROPHY.
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机译:肌营养不良蛋白的腺伴随病毒载体的递送,以治疗肌营养不良。
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摘要
The invention provides for recombinant AAV vectors comprising a miniaturized human micro-dystrophin gene and methods of using the recombinant vectors to reduce or prevent fibrosis in subjects suffering from muscular dystrophy.
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