Antisense oligonucleotide–directed inhibition of nonsense-mediated mRNA decay
| Autor: | Tomoki T. Nomakuchi, Isabel Aznarez, Adrian R. Krainer, Frank Rigo |
|---|---|
| Rok vydání: | 2015 |
| Předmět: |
0301 basic medicine
Nonsense-mediated decay Nonsense mutation Biomedical Engineering Bioengineering Applied Microbiology and Biotechnology Article 03 medical and health sciences Cell Line Tumor Sense (molecular biology) Humans Regulation of gene expression Chemistry Oligonucleotide Reproducibility of Results RNA Genetic Therapy Oligonucleotides Antisense Molecular biology Nonsense Mediated mRNA Decay 3. Good health Cell biology Antisense RNA 030104 developmental biology Gene Expression Regulation RNA splicing Molecular Medicine Biotechnology |
| Zdroj: | Nature biotechnology |
| ISSN: | 1546-1696 1087-0156 |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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