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

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,...

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Detalles Bibliográficos
Autores principales: Nomakuchi, Tomoki T., Rigo, Frank, Aznarez, Isabel, Krainer, Adrian R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2015
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4744113/
https://www.ncbi.nlm.nih.gov/pubmed/26655495
http://dx.doi.org/10.1038/nbt.3427
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author Nomakuchi, Tomoki T.
Rigo, Frank
Aznarez, Isabel
Krainer, Adrian R.
author_facet Nomakuchi, Tomoki T.
Rigo, Frank
Aznarez, Isabel
Krainer, Adrian R.
author_sort Nomakuchi, Tomoki T.
collection PubMed
description 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.
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spelling pubmed-47441132016-06-14 Antisense-oligonucleotide-directed inhibition of nonsense-mediated mRNA decay Nomakuchi, Tomoki T. Rigo, Frank Aznarez, Isabel Krainer, Adrian R. Nat Biotechnol Article 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. 2015-12-14 2016-02 /pmc/articles/PMC4744113/ /pubmed/26655495 http://dx.doi.org/10.1038/nbt.3427 Text en Users may view, print, copy, and download text and data-mine the content in such documents, for the purposes of academic research, subject always to the full Conditions of use: http://www.nature.com/authors/editorial_policies/license.html#terms
spellingShingle Article
Nomakuchi, Tomoki T.
Rigo, Frank
Aznarez, Isabel
Krainer, Adrian R.
Antisense-oligonucleotide-directed inhibition of nonsense-mediated mRNA decay
title Antisense-oligonucleotide-directed inhibition of nonsense-mediated mRNA decay
title_full Antisense-oligonucleotide-directed inhibition of nonsense-mediated mRNA decay
title_fullStr Antisense-oligonucleotide-directed inhibition of nonsense-mediated mRNA decay
title_full_unstemmed Antisense-oligonucleotide-directed inhibition of nonsense-mediated mRNA decay
title_short Antisense-oligonucleotide-directed inhibition of nonsense-mediated mRNA decay
title_sort antisense-oligonucleotide-directed inhibition of nonsense-mediated mrna decay
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4744113/
https://www.ncbi.nlm.nih.gov/pubmed/26655495
http://dx.doi.org/10.1038/nbt.3427
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