Chemistry, mechanism and clinical status of antisense oligonucleotides and duplex RNAs.

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Shen X, Corey DR

Chemistry, mechanism and clinical status of antisense oligonucleotides and duplex RNAs.

Nucleic Acids Res. 2018 Feb 28;46(4):1584-1600. doi: 10.1093/nar/gkx1239.

PubMed ID

29240946 [ View in PubMed

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Abstract

RNA plays a central role in the expression of all genes. Because any sequence within RNA can be recognized by complementary base pairing, synthetic oligonucleotides and oligonucleotide mimics offer a general strategy for controlling processes that affect disease. The two primary antisense approaches for regulating expression through recognition of cellular RNAs are single-stranded antisense oligonucleotides and duplex RNAs. This review will discuss the chemical modifications and molecular mechanisms that make synthetic nucleic acid drugs possible. Lessons learned from recent clinical trials will be summarized. Ongoing clinical trials are likely to decisively test the adequacy of our current generation of antisense nucleic acid technologies and highlight areas where more basic research is needed.

DrugBank Data that Cites this Article

Drugs

Inotersen

Drug Targets

Drug	Target	Kind	Organism	Pharmacological Action	Actions
Inotersen	Transthyretin mRNA	Nucleotide	Humans	Yes	Antisense oligonucleotide	Details