Invention Grant
- Patent Title: Gene therapy for ceroid lipofuscinoses
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Application No.: US16611512Application Date: 2018-05-11
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Publication No.: US11591614B2Publication Date: 2023-02-28
- Inventor: James M. Wilson , Nathan Katz , Christian Hinderer , Juliette Hordeaux
- 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 & Howson LLP
- Agent Colleen M. Schaller
- International Application: PCT/US2018/032278 WO 20180511
- International Announcement: WO2018/209205 WO 20181115
- Main IPC: C12N15/86
- IPC: C12N15/86 ; A61K48/00 ; C12N9/48 ; A61K9/00
Abstract:
Provided herein are methods and compositions for treatment of Batten disease. Such compositions include a recombinant adeno-associated virus (rAAV), said rAAV comprising an AAV capsid, and a vector genome packaged therein, said vector genome comprising (a) an AAV 5′ inverted terminal repeat (ITR) sequence; (b) a promoter; (c) a CLN2 coding sequence encoding a human TPP1; (d) an AAV 3′ ITR.
Public/Granted literature
- US20200239908A1 GENE THERAPY FOR CEROID LIPOFUSCINOSES Public/Granted day:2020-07-30
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