Gene therapy for treating mucopolysaccharidosis type II

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专利权人:

The Trustees of the University of Pennsyvania

发明人:

Hinderer, Christian,Wilson, James M.

申请号：

AU2017250298

公开号：

AU2017250298A1

申请日:

2017.04.14

申请国别(地区):

AU

年份:

2018

代理人:

摘要：

A suspension useful for AAV9-mediated intrathecal and/or systemic delivery of an expression cassette containing a hlDS gene is provided herein. Also provided are methods and kits containing these vectors and compositions useful for treating Hunter syndrome and the symptoms associated with Hunter syndrome.

来源网站：

中国工程科技知识中心

来源网址：

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