Invention Application
- Patent Title: GM3 SYNTHASE VECTORS AND USES THEREOF
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Application No.: US17602348Application Date: 2020-04-10
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Publication No.: US20220162571A1Publication Date: 2022-05-26
- Inventor: Guangping Gao , Huiya Yang , Kevin A. Strauss , Dan Wang
- Applicant: University of Massachusetts , Clinic for Special Children
- Applicant Address: US MA Boston; US PA Strasburg
- Assignee: University of Massachusetts,Clinic for Special Children
- Current Assignee: University of Massachusetts,Clinic for Special Children
- Current Assignee Address: US MA Boston; US PA Strasburg
- International Application: PCT/US20/27585 WO 20200410
- Main IPC: C12N9/10
- IPC: C12N9/10 ; C12N15/86 ; A61K48/00
Abstract:
Aspects of the disclosure relate to compositions and methods for expressing one or more Ganglioside GM3 synthase (GM3S) isoforms in a cell or subject. In some aspects, the disclosure relates to methods for treating GM3 synthase deficiency in a subject in need thereof.
Information query
