Invention Grant
- Patent Title: Methods and compositions for modulating sialic acid production and treating hereditary inclusion body myopathy
- Patent Title (中): 调节唾液酸生产和治疗遗传性包涵体肌病的方法和组合物
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Application No.: US12526239Application Date: 2008-02-07
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Publication No.: US09273300B2Publication Date: 2016-03-01
- Inventor: Phillip Maples , Chris Jay , John J. Nemunaitis
- Applicant: Phillip Maples , Chris Jay , John J. Nemunaitis
- Applicant Address: US TX Dallas
- Assignee: STRIKE BIO, INC
- Current Assignee: STRIKE BIO, INC
- Current Assignee Address: US TX Dallas
- Agency: Chalker Flores LLP
- Agent Edwin S. Flores
- International Application: PCT/US2008/001650 WO 20080207
- International Announcement: WO2008/097623 WO 20080814
- Main IPC: A61K48/00
- IPC: A61K48/00 ; A61K9/16 ; A61K9/50 ; C12N9/90 ; C12N15/85

Abstract:
According to certain embodiments of the present invention, methods for modulating the production of sialic acid in a system are provided, which comprise providing the system with a wild-type GNE-encoding nucleic acid sequence. According to such embodiments, the system may comprise a cell, muscular tissue, or other desirable targets. Similarly, the present invention encompasses methods for producing wild-type GNE in a system that comprises a mutated endogenous GNE-encoding sequence. In other words, the present invention includes providing, for example, a cell or muscular tissue that harbors a mutated (defective) GNE-encoding sequence with a functional wild-type GNE encoding sequence.
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