γ Peptide Nucleic Acid-Based miR-122 Inhibition Rescues Vascular Endothelial Dysfunction in Mice Fed a High-Fat Diet

J Med Chem. 2022 Feb 24;65(4):3332-3342. doi: 10.1021/acs.jmedchem.1c01831. Epub 2022 Feb 8.

Abstract

The blood levels of microRNA-122 (miR-122) is associated with the severity of cardiovascular disorders, and targeting it with efficient and safer miR inhibitors could be a promising approach. Here, we report the generation of a γ-peptide nucleic acid (γPNA)-based miR-122 inhibitor (γP-122-I) that rescues vascular endothelial dysfunction in mice fed a high-fat diet. We synthesized diethylene glycol-containing γP-122-I and found that its systemic administration counteracted high-fat diet (HFD)-feeding-associated increase in blood and aortic miR-122 levels, impaired endothelial function, and reduced glycemic control. A comprehensive safety analysis established that γP-122-I affects neither the complete blood count nor biochemical tests of liver and kidney functions during acute exposure. In addition, long-term exposure to γP-122-I did not change the overall adiposity, or histology of the kidney, liver, and heart. Thus, γP-122-I rescues endothelial dysfunction without any evidence of toxicity in vivo and demonstrates the suitability of γPNA technology in generating efficient and safer miR inhibitors.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adiposity / drug effects
  • Animals
  • Blood Cell Count
  • Blood Glucose / metabolism
  • Body Weight
  • Cardiovascular Diseases / drug therapy*
  • Diet, High-Fat
  • Drug Design
  • Endothelium, Vascular / drug effects*
  • Kidney Function Tests
  • Liver Function Tests
  • Mice
  • Mice, Inbred C57BL
  • MicroRNAs / antagonists & inhibitors*
  • MicroRNAs / blood
  • Muscle, Smooth, Vascular / drug effects
  • Peptide Nucleic Acids / adverse effects
  • Peptide Nucleic Acids / pharmacology*

Substances

  • Blood Glucose
  • MicroRNAs
  • Mirn122 microRNA, mouse
  • Peptide Nucleic Acids