Invention Grant
- Patent Title: Inhibiting or downregulating glycogen synthase by creating premature stop codons using antisense oligonucleotides
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Application No.: US15990463Application Date: 2018-05-25
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Publication No.: US10682423B2Publication Date: 2020-06-16
- Inventor: Carol A. Nelson , Bruce M. Wentworth , Ronald K. Scheule , Timothy E. Weeden , Nicholas P. Clayton
- Applicant: GENZYME CORPORATION
- Applicant Address: US NJ Summit
- Assignee: Genzyme Corporation
- Current Assignee: Genzyme Corporation
- Current Assignee Address: US NJ Summit
- Agency: Morrison & Foerster LLP
- Main IPC: A61K48/00
- IPC: A61K48/00 ; C07H21/04 ; C12N15/113

Abstract:
The present disclosure relates to antisense oligonucleotides (AONs) for modulating the expression of glycogen synthase. AONs of the present disclosure may be useful in treating diseases associated with the modulation of the expression of the enzyme glycogen synthase, such as Pompe disease. Also provided by the present disclosure are compositions comprising AONs, as well as methods of down regulating mRNA coding for glycogen synthase, methods for reducing glycogen synthase in skeletal and cardiac muscle, and methods for treating Pompe disease.
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