MicroRNA-based therapeutics in cardiovascular disease: screening and delivery to the target

Biochem Soc Trans. 2018 Feb 19;46(1):11-21. doi: 10.1042/BST20170037. Epub 2017 Dec 1.

Abstract

MicroRNAs (miRNAs) are small non-coding RNAs of ∼22 nucleotides, which have increasingly been recognized as potent post-transcriptional regulators of gene expression. MiRNA targeting is defined by the complementarities between positions 2-8 of miRNA 5'-end with generally the 3'-untranslated region of target mRNAs (messenger RNAs). The capacity of miRNAs to simultaneously inhibit many different mRNAs allows for an amplification of biological responses. Hence, miRNAs are extremely attractive targets for therapeutic regulation in several diseases, including cardiovascular. Novel approaches are emerging to identify the miRNA functions in cardiovascular biology processes and to improve miRNA delivery in the heart and vasculature. In the present study, we provide an overview of current studies of miRNA functions in cardiovascular cells by the use of high-content screening. We also discuss the challenge to achieve a safe and targeted delivery of miRNA therapeutics in cardiovascular cells.

Keywords: cardiovascular disease; gene therapy; high-throughput screening; microRNA.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • 3' Untranslated Regions
  • Cardiovascular Diseases / drug therapy*
  • Drug Delivery Systems*
  • Humans
  • MicroRNAs / genetics
  • MicroRNAs / metabolism
  • MicroRNAs / therapeutic use*
  • Myocardium / metabolism
  • RNA, Messenger / genetics

Substances

  • 3' Untranslated Regions
  • MicroRNAs
  • RNA, Messenger