AAV9-mediated gene therapy for treating mucopolysaccharidosis type I

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专利权人:

The Trustees of the University of Pennsylvania

发明人:

HINDERER, Christian,WILSON, James M.

申请号：

AU2018298133

公开号：

AU2018298133A1

申请日:

2018.07.05

申请国别(地区):

AU

年份:

2020

代理人:

摘要：

A co-therapeutic regimen comprising AAV9-mediated intrathecal/intracisternal and/or systemic delivery of an expression cassette containing a hIDUA gene and two or more immunosuppressants is provided herein. Also provided are methods useful for treating hIDUA deficiency (MPSI) and the symptoms associated with Hurler, Hurler-Scheie and Scheie syndromes.

来源网站：

中国工程科技知识中心

来源网址：

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