Invention Grant
- Patent Title: Adeno-associated virus vectors for treatment of glycogen storage disease
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Application No.: US15038979Application Date: 2014-11-25
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Publication No.: US09644216B2Publication Date: 2017-05-09
- Inventor: Janice J. Chou , Barry J. Byrne
- Applicant: The United States of America, as represented by the Secretary, Department of Health and Human Services , University of Florida Research Foundation, Incorporated
- Applicant Address: US DC Washington US FL Gainesville
- Assignee: The United States of America, as represented by the Secretary, Department of Health and Human Services,University of Florida Research Foundation, Incorporated
- Current Assignee: The United States of America, as represented by the Secretary, Department of Health and Human Services,University of Florida Research Foundation, Incorporated
- Current Assignee Address: US DC Washington US FL Gainesville
- Agency: Klarquist Sparkman, LLP
- International Application: PCT/US2014/067415 WO 20141125
- International Announcement: WO2015/081101 WO 20150604
- Main IPC: C12N15/63
- IPC: C12N15/63 ; C12N5/00 ; C12P21/06 ; C12N15/86 ; C12N7/00 ; C12N9/16 ; A61K48/00

Abstract:
The present disclosure describes improved adeno-associated virus (AAV) vectors for gene therapy applications in the treatment of glycogen storage disease, particularly glycogen storage disease type Ia (GSD-Ia). Described are recombinant nucleic acid molecules, vectors and recombinant AAV that include a G6PC promoter/enhancer, a synthetic intron, a G6PC coding sequence (such as a wild-type or codon-optimized G6PC coding sequence), and stuffer nucleic acid sequence situated between the G6PC promoter/enhancer and the intron, as well as between the intron and the G6PC coding sequence. The recombinant AAVs disclosed herein exhibit highly efficient liver transduction and are capable of correcting metabolic abnormalities in an animal model of GSD-Ia.
Public/Granted literature
- US20160376608A1 ADENO-ASSOCIATED VIRUS VECTORS FOR TREATMENT OF GLYCOGEN STORAGE DISEASE Public/Granted day:2016-12-29
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