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Inhibiting or downregulating glycogen synthase by creating premature stop codons using antisense oligonucleotides

a technology of antisense oligonucleotides and antisense oligonucleotides, which is applied in the direction of transferases, drug compositions, metabolic disorders, etc., can solve the problems of insufficient treatment, serious breathing problems, and inability to completely eliminate the treatment method, so as to suppress inhibit the biosynthesis of glycogen , the effect of reducing the accumulation of glycogen in muscle tissu

Inactive Publication Date: 2017-06-29
GENZYME CORP
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Benefits of technology

The patent aims to treat symptoms caused by the buildup of glycogen in the body's cells by controlling the expression of glycogen synthase. By reducing the amount of glycogen synthase in muscle tissue, the invention can decrease glycogen synthase enzyme activity and reduce the buildup of glycogen. This approach may offer a beneficial effect for mammals afflicted with glycogen buildup-related symptoms.

Problems solved by technology

Serious breathing problems can occur and children with this form of Pompe disease do not live past early childhood.
However, people with this form of the disease experience progressive muscle weakness, which can lead to breathing problems and may eventually lead to respiratory failure.
However, this method of treatment is not always entirely effective, and as a result, additional therapies for Pompe disease are needed.

Method used

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  • Inhibiting or downregulating glycogen synthase by creating premature stop codons using antisense oligonucleotides
  • Inhibiting or downregulating glycogen synthase by creating premature stop codons using antisense oligonucleotides
  • Inhibiting or downregulating glycogen synthase by creating premature stop codons using antisense oligonucleotides

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Methods and Materials

[0080]Design of Phosphorodiamidate Morpholino Oligomers

[0081]Phosphorodiamidate morpholino oligomers (PMOs) were designed to hybridize to Gys1 mRNA so as to invoke either exon skipping or translation inhibition as described by Morcos. The sequences designed to skip exons in Gys1 mRNA are as follows:

[0082]PMO 1 (5′-TCAGGGTTGTGGACTCAATCATGCC-3′) (SEQ ID NO.: 54) targeted the intronic sequence proximal to the splice acceptor site of intron 7;

[0083]PMO 2 (5′-AAGGACCAGGGTAAGACTAGGGACT-3′) (SEQ ID NO.: 55) targeted the intronic sequence proximal to the splice acceptor site of intron 4;

[0084]PMO 3 (5′-GTCCTGGACAAGGATTGCTGACCAT-3′) (SEQ ID NO.: 56) targeted the exon-intron boundary of exon 8;

[0085]PMO 4 (5′-CTGCTTCCTTGTCTACATTGAACTG-3′) (SEQ ID NO.: 57) targeted the intron-exon boundary of exon 5;

[0086]PMO 5 (5′-ATACCCGGCCCAGGTACTTCCAATC-3′) (SEQ ID NO.: 58) targeted the exon-intron boundary of exon 14;

[0087]PMO 6 (5′-CTGGACAAGGATTGCTGACCATAGT-3′) (SEQ ID NO.: 59), simi...

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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.

Description

CROSS-REFERENCE TO RELATED APPLICATION[0001]This application claims the benefit of priority to U.S. Provisional Patent Application No. 62 / 002,294, filed May 23, 2014, which is hereby incorporated by reference in its entirety.DESCRIPTION OF THE INVENTION[0002]Field of the Invention[0003]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.SUMMARY OF THE INVENTION[0004]Pompe disease is an inherited disorder caused by the accumulation of glycogen in the body's cells. This buildup of glycogen in the b...

Claims

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Application Information

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IPC IPC(8): A61K48/00C12N15/113
CPCA61K48/005C12N15/1137C12N2310/3233C12N2320/33C12N2310/3513C12N2310/11C12Y204/01011A61P3/10C12N15/113
Inventor NELSON, CAROL A.WENTWORTH, BRUCE M.SCHEULE, RONALD K.WEEDEN, TIMOTHY E.CLAYTON, NICHOLAS P.
Owner GENZYME CORP
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