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

申请号：

SG11201808176T

公开号：

SG11201808176TA

申请日:

2017.04.14

申请国别(地区):

SG

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.

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中国工程科技知识中心

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