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Published on: September 21, 2017

Antisense oligonucleotides for the treatment of dyslipidemia

I Gouni-Berthold1, H K Berthold

  • 1Department of Internal Medicine II, University of Cologne, Cologne, Germany. ioanna.berthold@uni-koeln.de

Current Pharmaceutical Design
|March 23, 2011
PubMed

Insights

New antisense oligonucleotide therapies show promise for lowering low-density lipoprotein (LDL)-cholesterol, addressing challenges in cardiovascular disease (CVD) risk management when current medications fall short.

Area of Science:

  • Pharmacology
  • Genetics
  • Cardiovascular Medicine

Background:

  • Lowering low-density lipoprotein (LDL)-cholesterol reduces cardiovascular disease (CVD) risk.
  • Current guidelines recommend increasingly lower LDL-cholesterol targets.
  • Existing therapies, including statins, may not be sufficient for all patients to reach target LDL-cholesterol levels.

Purpose of the Study:

  • To review the efficacy of antisense oligonucleotides (ASO) for treating dyslipidemia.
  • To focus on ASO targeting apolipoprotein B-100 (ApoB-100) and proprotein convertase subtilisin/kexin type 9 (PCSK9).

Main Methods:

  • Review of available data on ASO therapies.
  • Focus on ASO mechanisms involving base-pair hybridization to reduce target protein production.
  • Examination of genetic models in mice and humans demonstrating the link between reduced ApoB-100 and PCSK9 and lower LDL-cholesterol.

Main Results:

  • Antisense oligonucleotides (ASO) offer a novel therapeutic strategy for dyslipidemia.
  • Decreasing the production of hepatic apolipoprotein B-100 (ApoB-100) and PCSK9 lowers circulating LDL-cholesterol.
  • ASO targeting ApoB-100 are advanced in clinical development.

Conclusions:

  • Antisense oligonucleotides represent a new class of drugs for managing dyslipidemia.
  • Targeting ApoB-100 and PCSK9 with ASO provides a promising approach to lower LDL-cholesterol.
  • Further development of ASO is warranted to address unmet needs in CVD prevention.

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