Neurodegenerative Disorders Treatment: The MicroRNA Role

Curr Gene Ther. 2017;17(5):327-363. doi: 10.2174/1566523218666180119120726.


Neurodegenerative diseases such as Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis, Huntington's disease and prion disease are not timely and effectively treated using conventional therapies. This emphasizes the need for alternative therapeutic approaches. In this respect, gene-based therapies have been adopted as potentially feasible alternative therapies, where the microRNA (miRNA) approach has experienced a great explosion in recent years. Because miRNAs have been shown to be implicated in the pathogenesis of several diseases including neurodegenerative diseases, they are intensely studied as candidates for diagnostic and prognostic biomarkers, as predictors of drug response and as therapeutic agents. In this review, we evaluate the feasibility of both direct and indirect miRNA mimics and inhibitors toward the regulation of neurodegenerative-related genes both in vivo and in vitro models, highlight the advantages and drawbacks associated with miRNA-based therapy, and summarize the relevant techniques and approaches attempted to deliver miRNAs to the central nervous system for therapeutic purposes, with particular regard to the exosomes. Additionally, we describe a new approach that holds great promise for the treatment of a wide range of diseases including neurodegenerative disorders. This approach is based on addressing the incorporation of miRNAs into exosomes to increase the quantity and quality of miRNA packed and delivered to the central nervous system and other sites of action.

Keywords: Exosomes; MicroRNA; Neurodegenerative diseases; Therapy.; miRNA delivery; miRNA inhibitors; miRNA mimics; miRNA-based therapy.

Publication types

  • Review

MeSH terms

  • Alzheimer Disease / genetics
  • Alzheimer Disease / therapy
  • Amyotrophic Lateral Sclerosis / genetics
  • Amyotrophic Lateral Sclerosis / therapy
  • Exosomes / genetics*
  • Gene Expression Regulation
  • Genetic Therapy / methods*
  • Genetic Therapy / trends
  • Humans
  • MicroRNAs / genetics*
  • Neurodegenerative Diseases / genetics
  • Neurodegenerative Diseases / therapy*
  • Parkinson Disease / genetics
  • Parkinson Disease / therapy
  • RNA Interference


  • MicroRNAs