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Antisense oligonucleotide-directed inhibition of nonsense-mediated mRNA decay.

ABSTRACT: Nonsense-mediated mRNA decay (NMD) is a cellular quality-control mechanism that is thought to exacerbate the phenotype of certain pathogenic nonsense mutations by preventing the expression of semi-functional proteins. NMD also limits the efficacy of read-through compound (RTC)-based therapies. Here, we report a gene-specific method of NMD inhibition using antisense oligonucleotides (ASOs) and combine this approach with an RTC to effectively restore the expression of full-length protein from a nonsense-mutant allele.

SUBMITTER: Nomakuchi TT

PROVIDER: S-EPMC4744113 | biostudies-literature | 2016 Feb

REPOSITORIES: biostudies-literature

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Antisense oligonucleotide-directed inhibition of nonsense-mediated mRNA decay.

Nomakuchi Tomoki T TT Rigo Frank F Aznarez Isabel I Krainer Adrian R AR

Nature biotechnology 20151214 2

Nonsense-mediated mRNA decay (NMD) is a cellular quality-control mechanism that is thought to exacerbate the phenotype of certain pathogenic nonsense mutations by preventing the expression of semi-functional proteins. NMD also limits the efficacy of read-through compound (RTC)-based therapies. Here, we report a gene-specific method of NMD inhibition using antisense oligonucleotides (ASOs) and combine this approach with an RTC to effectively restore the expression of full-length protein from a no ...[more]

PMID: 26655495

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