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Article

Attenuation of Nonsense-Mediated mRNA Decay Enhances In Vivo Nonsense Suppression

10 Apr 2013

Abstract excerpt

Nonsense suppression therapy is an approach to treat genetic diseases caused by nonsense mutations. This therapeutic strategy pharmacologically suppresses translation termination at Premature Termination Codons (PTCs) in order to restore expression of functional protein. However, the process of Nonsense-Mediated mRNA Decay (NMD), which reduces the abundance of mRNAs containing PTCs, frequently limits this...

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PMID: 23593225
DOI: 10.1371/journal.pone.0060478

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