Reversal of siRNA-mediated gene silencing in vivo.
basic_science · Level V
Where this comes from
- Record sourced from PubMed, PMID 29786096.
- Also identified by DOI 10.1038/nbt.4136.
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Abstract
We report rapid, potent reversal of GalNAc-siRNA-mediated RNA interference (RNAi) activity in vivo with short, synthetic, high-affinity oligonucleotides complementary to the siRNA guide strand. We found that 9-mers with five locked nucleic acids (LNAs) have the highest potency across several targets. Our modular, sequence-specific approach, named REVERSIR, may enhance the therapeutic profile of any long-acting GalNAc-siRNA (short interfering RNA) conjugate by enabling control of RNAi pharmacology.
Medical subject headings
- Gene Silencing
- RNA, Small Interfering