Current Status of microRNA-Based Therapeutic Approaches in Neurodegenerative Disorders

Cells. 2020 Jul 15;9(7):1698. doi: 10.3390/cells9071698.

Abstract

MicroRNAs (miRNAs) are a key gene regulator and play essential roles in several biological and pathological mechanisms in the human system. In recent years, plenty of miRNAs have been identified to be involved in the development of neurodegenerative disorders (NDDs), thus making them an attractive option for therapeutic approaches. Hence, in this review, we provide an overview of the current research of miRNA-based therapeutics for a selected set of NDDs, either for their high prevalence or lethality, such as Alzheimer's, Parkinson's, Huntington's, Amyotrophic Lateral Sclerosis, Friedreich's Ataxia, Spinal Muscular Atrophy, and Frontotemporal Dementia. We also discuss the relevant delivery techniques, pertinent outcomes, their limitations, and their potential to become a new generation of human therapeutic drugs in the near future.

Keywords: MicroRNA; miRNA delivery; miRNA therapeutics; neurodegenerative disorders.

Publication types

  • Review

MeSH terms

  • Animals
  • Gene Transfer Techniques
  • Humans
  • MicroRNAs / biosynthesis
  • MicroRNAs / genetics
  • MicroRNAs / therapeutic use*
  • Models, Biological
  • Neurodegenerative Diseases / therapy*

Substances

  • MicroRNAs