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Article

A regulated NMD mouse model supports NMD inhibition as a viable therapeutic option to treat genetic diseases.

Disease models & mechanisms - 27 Aug 2020

Echols Josh, Siddiqui Amna, Dai Yanying, Havasi Viktoria, Sun Richard, Kaczmarczyk Aneta, Keeling Kim M

Abstract excerpt

Nonsense-mediated mRNA decay (NMD) targets mRNAs that contain a premature termination codon (PTC) for degradation, preventing their translation. By altering the expression of PTC-containing mRNAs, NMD modulates the inheritance pattern and severity of genetic diseases. NMD also limits the efficiency of suppressing translation termination at PTCs, an emerging therapeutic approach to treat genetic diseases caused by...

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Identifiers

PMID: 32737261
DOI: 10.1242/dmm.044891
PMCID: PMC7473645

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