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)
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
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| 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 Cancer Center Program > Gene Regulation and Cell Proliferation
CSHL Cancer Center Program > Gene Regulation and Inheritance Program
CSHL labs > Krainer lab
CSHL Cancer Center Shared Resources > Antibody and Phage Display Service |
| Depositing User: |
Matt Covey
|
| Date: |
February 2016 |
| Date Deposited: |
22 Feb 2016 16:59 |
| Last Modified: |
09 Nov 2020 21:17 |
| PMCID: |
PMC4744113 |
| Related URLs: |
|
| URI: |
https://repository.cshl.edu/id/eprint/32365 |
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