Strategies to use microRNAs as therapeutic targets

Best Pract Res Clin Endocrinol Metab. 2016 Oct;30(5):551-561. doi: 10.1016/j.beem.2016.07.004. Epub 2016 Aug 1.


MicroRNAs (miRNAs) provide a unique mechanism of gene regulation and play a key role in different pathologies ranging from metabolic diseases to cancer. miRNAs can impact biological function as either suppressors of gene expression when their expression levels are enhanced in a disease state or they can cause upregulation of gene expression when their expression levels are reduced. Therefore both gain- and loss-of- function strategies are needed to fully exploit their therapeutic potential. miRNA research first focused on inhibition of single miRNAs using oligonucleotide inhibitors. However, more recent approaches explore the potential to deliver oligonucleotides to mimic miRNA expression or to employ small molecules to increase or inhibit miRNA function. Although we need to know more about the potential side effects and tissue specific delivery systems, these studies provide grounds to further exploit miRNAs as novel therapeutic targets in the clinic.

Keywords: cancer; metabolism; microRNA; nanoparticles; oligonucleotide inhibitors; small molecules.

Publication types

  • Review

MeSH terms

  • Animals
  • Gene Silencing
  • Genetic Therapy / methods*
  • Humans
  • Metabolic Diseases / genetics
  • Metabolic Diseases / therapy*
  • MicroRNAs / drug effects
  • MicroRNAs / genetics*
  • MicroRNAs / metabolism
  • Neoplasms / genetics
  • Neoplasms / therapy*
  • Oligonucleotides / pharmacology
  • Oligonucleotides / therapeutic use


  • MicroRNAs
  • Oligonucleotides