Invention Grant
- Patent Title: Gene therapy for treating mucopolysaccharidosis type II
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Application No.: US16093413Application Date: 2017-04-14
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Publication No.: US11253612B2Publication Date: 2022-02-22
- Inventor: Christian Hinderer , James M. Wilson
- Applicant: The Trustees of the University of Pennsylvania
- Applicant Address: US PA Philadelphia
- Assignee: The Trustees of the University of Pennsylvania
- Current Assignee: The Trustees of the University of Pennsylvania
- Current Assignee Address: US PA Philadelphia
- Agency: Howson and Howson LLP
- Agent Cathy Kodroff
- International Application: PCT/US2017/027770 WO 20170414
- International Announcement: WO2017/181113 WO 20171019
- Main IPC: A61K48/00
- IPC: A61K48/00 ; A61P3/00 ; C12N15/113 ; C12N15/85 ; C12N9/16 ; C12Q1/6827 ; C12N15/86
Abstract:
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.
Public/Granted literature
- US20190070311A1 GENE THERAPY FOR TREATING MUCOPOLYSACCHARIDOSIS TYPE II Public/Granted day:2019-03-07
Information query
