THERAPIE GENIQUE MEDIEE PAR AAV9 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：

CA3068328

公开号：

CA3068328A1

申请日:

2018.07.05

申请国别(地区):

CA

年份:

2019

代理人:

摘要：

A co-therapeutic regimen comprising AAV9-mediated intrathecal/intracisternaland/or systemic delivery of anexpression cassette containing a hIDUA gene and two or more immunosuppressantsis provided herein. Also provided are methods useful fortreating 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

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