METHODS AND MATERIALS FOR GALGT2 GENE THERAPY

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专利权人:

发明人:

Paul Taylor Martin

申请号：

US15759474

公开号：

US20180250423A1

申请日:

2016.09.16

申请国别(地区):

US

年份:

2018

代理人:

摘要：

The present disclosure relates to recombinant adeno-associated virus (rAAV) delivery of a GALGT2 polynucleotide. The disclosure provides rAAV and methods of using the rAAV for GALGT2 gene therapy of neuromuscular disorders. Exemplary neuromuscular disorders include, but are not limited to, muscular dystrophies such as Duchenne muscular dystrophy, Congenital Muscular Dystrophy 1A and Limb Girdle Muscular Dystrophy 2D.

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中国工程科技知识中心

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