Inhibition of miR-33a/b in non-human primates raises plasma HDL and lowers VLDL triglycerides

Nature. 2011 Oct 19;478(7369):404-7. doi: 10.1038/nature10486.

Abstract

Cardiovascular disease remains the leading cause of mortality in westernized countries, despite optimum medical therapy to reduce the levels of low-density lipoprotein (LDL)-associated cholesterol. The pursuit of novel therapies to target the residual risk has focused on raising the levels of high-density lipoprotein (HDL)-associated cholesterol in order to exploit its atheroprotective effects. MicroRNAs (miRNAs) have emerged as important post-transcriptional regulators of lipid metabolism and are thus a new class of target for therapeutic intervention. MicroRNA-33a and microRNA-33b (miR-33a/b) are intronic miRNAs whose encoding regions are embedded in the sterol-response-element-binding protein genes SREBF2 and SREBF1 (refs 3-5), respectively. These miRNAs repress expression of the cholesterol transporter ABCA1, which is a key regulator of HDL biogenesis. Recent studies in mice suggest that antagonizing miR-33a may be an effective strategy for raising plasma HDL levels and providing protection against atherosclerosis; however, extrapolating these findings to humans is complicated by the fact that mice lack miR-33b, which is present only in the SREBF1 gene of medium and large mammals. Here we show in African green monkeys that systemic delivery of an anti-miRNA oligonucleotide that targets both miR-33a and miR-33b increased hepatic expression of ABCA1 and induced a sustained increase in plasma HDL levels over 12 weeks. Notably, miR-33 antagonism in this non-human primate model also increased the expression of miR-33 target genes involved in fatty acid oxidation (CROT, CPT1A, HADHB and PRKAA1) and reduced the expression of genes involved in fatty acid synthesis (SREBF1, FASN, ACLY and ACACA), resulting in a marked suppression of the plasma levels of very-low-density lipoprotein (VLDL)-associated triglycerides, a finding that has not previously been observed in mice. These data establish, in a model that is highly relevant to humans, that pharmacological inhibition of miR-33a and miR-33b is a promising therapeutic strategy to raise plasma HDL and lower VLDL triglyceride levels for the treatment of dyslipidaemias that increase cardiovascular disease risk.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • ATP Binding Cassette Transporter 1
  • ATP-Binding Cassette Transporters / metabolism
  • Animals
  • Cells, Cultured
  • Chlorocebus aethiops* / blood
  • Chlorocebus aethiops* / genetics
  • Chlorocebus aethiops* / metabolism
  • Cholesterol, LDL / blood
  • Gene Expression Regulation / drug effects*
  • Gene Silencing
  • HEK293 Cells
  • Humans
  • Lipoproteins, HDL / blood*
  • Lipoproteins, VLDL / blood*
  • Liver / drug effects*
  • Liver / metabolism
  • Male
  • MicroRNAs / antagonists & inhibitors*
  • MicroRNAs / metabolism
  • Oligoribonucleotides, Antisense / pharmacology*
  • Time Factors
  • Triglycerides / blood*

Substances

  • ABCA1 protein, human
  • ATP Binding Cassette Transporter 1
  • ATP-Binding Cassette Transporters
  • Cholesterol, LDL
  • Lipoproteins, HDL
  • Lipoproteins, VLDL
  • MicroRNAs
  • Oligoribonucleotides, Antisense
  • Triglycerides
  • very low density lipoprotein triglyceride

Associated data

  • GEO/GSE31177