Gene therapy for treating mucopolysaccharidosis type i

• 收藏
• 分享

专利权人:

The Trustees of the University of Pennsylvania

发明人:

Hinderer, Christian,Wilson, James M.

申请号：

AU2017215211

公开号：

AU2017215211A1

申请日:

2017.02.02

申请国别(地区):

AU

年份:

2018

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/