THERAPIE GENIQUE POUR TRAITER LA MUCOPOLYSACCHARIDOSE DE 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：

CA3012195

公开号：

CA3012195A1

申请日:

2017.02.02

申请国别(地区):

CA

年份:

2017

代理人:

摘要：

A suspension useful for AAV9-mediated intrathecal/intracisternal and/or systemic delivery of an expression cassette containing a hIDUA gene is provided herein. Also provided are methods and kits containing these vectors and compositions useful for treating 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/