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10.1038/nbt.3427

http://scihub22266oqcxt.onion/10.1038/nbt.3427
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C4744113!4744113!26655495
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suck abstract from ncbi


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pmid26655495      Nat+Biotechnol 2016 ; 34 (2): 164-6
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  • Antisense-oligonucleotide-directed inhibition of nonsense-mediated mRNA decay #MMPMID26655495
  • Nomakuchi TT; Rigo F; Aznarez I; Krainer AR
  • Nat Biotechnol 2016[Feb]; 34 (2): 164-6 PMID26655495show ga
  • 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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