COMPOSITIONS USEFUL FOR TREATING GM1 GANGLIOSIDOSIS

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专利权人:

The Trustees of The University of Pennsylvania

发明人:

申请号：

EP21711072.5

公开号：

EP4096721A1

申请日:

2021.02.01

申请国别(地区):

EP

年份:

2022

代理人:

摘要：

A therapeutic regimen useful for treatment of GM1 gangliosidosis comprising administration of a recombinant adeno-associated virus (rAAV) vector having an AAV capsid and a vector genome comprising a sequence encoding human β-galactosidase is provided. Also provided are compositions containing a rAAV vector and methods of treating GM1 gangliosidosis in patient comprising administration of a rAAV vector.

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中国工程科技知识中心

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http://www.ckcest.cn/home/