Inhibition of nonsense mediated mRNA decay by drugs that prevent hypusination of eukaryotic initiation factor 5a

a technology of eukaryotic initiation factor and mrna decay, which is applied in the direction of pharmaceutical active ingredients, organic active ingredients, medical preparations, etc., can solve the problems of no biological activity, low biological activity of prematurely terminated proteins, and even harmful to biological functions
US20170319612A9Active Publication Date: 2017-11-09RUTGERS THE STATE UNIV

Patent Information

Authority / Receiving Office
US · United States
Patent Type
Applications(United States)
Current Assignee / Owner
RUTGERS THE STATE UNIV
Publication Date
2017-11-09

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Abstract

Provided are methods for treating a NAD comprising administering to a patient suffering from a NAD an inhibitor of NMD and a nonsense suppressor, whereby degradation of NMD susceptible mRNA is decreased and translation termination at a misplaced nonsense codon is blocked.
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Description

CROSS REFERENCE TO RELATED APPLICATIONS

[0001] The instant application claims priority under 35 U.S.C. §119(e) to U.S. Provisional Application No. 61 / 496,378, filed Jun. 13, 2012, and is also a continuation-in-part of U.S. application Ser. No. 12 / 840,270, filed Jul. 20, 2010, which claims 35 U.S.C. §119(e) priority to U.S. Provisional Application No. 61 / 271,409, filed Jul. 20, 2009, all of which are incorporated by reference in their entirety.FIELD OF THE INVENTION

[0002] The present invention relates to methods of treating nonsense-associated diseases and inhibiting nonsense-mediated mRNA decay by administering an inhibitor of NMD and a nonsense suppressor.BACKGROUND OF THE INVENTION

[0003] Nonsense-mediated decay (“NMD”) is a cellular mechanism that selectively degrades faulty messenger RNA (“mRNA”) containing an out-of-place stop (nonsense) codon. If translated, such mRNAs would produce a shortened version of the encoded protein. The NMD surveillance mechanism reduces or prevents the f...

Claims

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