- 专利标题： AAV9-MEDIATED GENE THERAPY FOR TREATING MUCOPOLYSACCHARIDOSIS TYPE I
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申请号： US18538866申请日： 2023-12-13
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公开(公告)号： US20240307511A1公开(公告)日： 2024-09-19
- 发明人: Christian Hinderer , James M. Wilson
- 申请人： The Trustees of the University of Pennsylvania
- 申请人地址： US PA Philadelphia
- 专利权人： The Trustees of the University of Pennsylvania
- 当前专利权人： The Trustees of the University of Pennsylvania
- 当前专利权人地址： US PA Philadelphia
- 主分类号： A61K38/47
- IPC分类号： A61K38/47 ; A61K9/00 ; A61K31/343 ; A61K31/439 ; A61K35/76 ; A61K45/06 ; A61K48/00 ; A61P3/00 ; C12N7/00 ; C12N9/24 ; C12N15/86
摘要：
A co-therapeutic regimen comprising AAV9-mediated intrathecal/intracisternal and/or systemic delivery of an expression cassette containing a hIDUA gene and two or more immunosuppressants is provided herein. Also provided are methods useful for treating hIDUA deficiency (MPSI) and the symptoms associated with Hurler, Hurler-Scheie and Scheie syndromes.
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