Antisense oligonucleotide-directed inhibition of nonsense-mediated mRNA decay.
basic_science · Level V
Where this comes from
- Record sourced from PubMed, PMID 26655495.
- Also identified by DOI 10.1038/nbt.3427 and PMC identifier 4744113.
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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.
Medical subject headings
- Gene Expression Regulation
- Genetic Therapy
- Nonsense Mediated mRNA Decay
- Oligonucleotides, Antisense