Antisense technology: an emerging platform for cardiovascular disease therapeutics

J Cardiovasc Transl Res. 2013 Dec;6(6):969-80. doi: 10.1007/s12265-013-9495-7. Epub 2013 Jul 16.


Antisense oligonucleotides and small interfering RNAs, which suppress the translation of specific mRNA target proteins, are emerging as important therapeutic modalities for the treatment of cardiovascular disease. Over the last 25 years, the advances in all aspects of antisense technology, as well as a detailed understanding of the mechanism of action of antisense drugs, have enabled their use as therapeutic agents. These advancements culminated in the FDA approval of the first chronically administered cardiovascular antisense therapeutic, mipomersen, which targets hepatic apolipoprotein B mRNA. This review provides a brief history of antisense technology, highlights the progression of mipomersen from preclinical studies to multiple Phase III registration trials, and gives an update on the status of other cardiovascular antisense therapeutics currently in the clinic.

Publication types

  • Review

MeSH terms

  • Animals
  • Anticholesteremic Agents / therapeutic use
  • Apolipoproteins B / genetics
  • Apolipoproteins B / metabolism
  • Cardiovascular Diseases / genetics
  • Cardiovascular Diseases / metabolism
  • Cardiovascular Diseases / therapy*
  • Dyslipidemias / genetics
  • Dyslipidemias / metabolism
  • Dyslipidemias / therapy
  • Gene Expression Regulation
  • Genetic Therapy / methods*
  • Humans
  • Liver / metabolism
  • Oligonucleotides / therapeutic use
  • Oligonucleotides, Antisense / therapeutic use*
  • RNA, Messenger / metabolism


  • Anticholesteremic Agents
  • Apolipoproteins B
  • Oligonucleotides
  • Oligonucleotides, Antisense
  • RNA, Messenger
  • mipomersen