ריפוי גני לטיפול במחלת-רב-סוכר רירי סוג ii

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专利权人:

THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA

发明人:

申请号：

IL26221118

公开号：

IL262211D0

申请日:

2018.10.08

申请国别(地区):

IL

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 hIDS 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/