Antisense oligonucleotide-directed inhibition of nonsense-mediated mRNA decay

Nomakuchi, T. T., Rigo, F., Aznarez, I., Krainer, A. R. (February 2016) Antisense oligonucleotide-directed inhibition of nonsense-mediated mRNA decay. Nat Biotechnol, 34 (2). pp. 164-166. ISSN 1546-1696 (Electronic)1087-0156 (Linking)

URL: http://www.ncbi.nlm.nih.gov/pubmed/26655495
DOI: 10.1038/nbt.3427

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.

Item Type: Paper
Subjects: bioinformatics > genomics and proteomics > genetics & nucleic acid processing > DNA, RNA structure, function, modification
bioinformatics > genomics and proteomics > genetics & nucleic acid processing > DNA, RNA structure, function, modification > antisense
bioinformatics > genomics and proteomics > genetics & nucleic acid processing > DNA, RNA structure, function, modification > oligonucleotide
bioinformatics > genomics and proteomics > genetics & nucleic acid processing > DNA, RNA structure, function, modification > RNA splicing
CSHL Authors:
Communities: CSHL labs > Krainer lab
CSHL Cancer Center Program > Gene Regulation and Cell Proliferation
Depositing User: Matt Covey
Date: February 2016
Date Deposited: 22 Feb 2016 16:59
Last Modified: 06 Mar 2018 16:40
PMCID: PMC4744113
Related URLs:
URI: http://repository.cshl.edu/id/eprint/32365

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