Adeno-associated viral vectors useful in treatment of spinal muscular atropy

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专利权人:

The Trustees of the University of Pennsylvania

发明人:

Wilson, James M.,Hinderer, Christain

申请号：

AU2016370630

公开号：

AU2016370630A1

申请日:

2016.12.14

申请国别(地区):

AU

年份:

2018

代理人:

摘要：

Compositions and methods useful in treating spinal muscular atrophy are provided. The compositions comprise a recombinant adeno-associated viral vector containing an AAV capsid, e.g., AAVrh.10 capsid, and nucleic acid sequences encoding a functional SMN protein. The methods involve administering these compositions to humans in need thereof.

来源网站：

中国工程科技知识中心

来源网址：

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