The therapeutic potential of microRNAs in nervous system damage, degeneration, and repair

Neuromolecular Med. 2009;11(3):153-61. doi: 10.1007/s12017-009-8086-x.

Abstract

MicroRNAS (miRNAs) have been suggested to play important roles in the central nervous system during development as well as disease. miRNAs appear to be dysregulated in a number of neurodegenerative diseases, developmental disorders, and as a result of stroke. Each miRNA has the ability to regulate hundreds of messenger RNA transcripts, both by causing degradation of the mRNA and by inhibition of protein translation. Recent findings suggest that it may eventually be possible to treat some neurological disorders by restoring or inhibiting miRNAs altered by disease pathology. Both viral delivery and administration of modified oligonucleotides mimicking or inhibiting specific miRNAs have been effective in model systems. Artificial miRNAs have also been generated for the repression of specific transcripts. Alteration of miRNA expression by disease and insult also holds the potential for improved diagnostic tools. Finally, miRNAs have been shown to control cellular proliferation and specification, suggesting that manipulation of miRNAs in cultured cells could result in more convenient generation of pure cell populations for transplantation.

MeSH terms

  • Animals
  • Central Nervous System* / pathology
  • Central Nervous System* / physiology
  • Central Nervous System* / physiopathology
  • Gene Expression Regulation
  • Humans
  • MicroRNAs* / genetics
  • MicroRNAs* / metabolism
  • MicroRNAs* / therapeutic use
  • Nerve Regeneration / physiology*
  • Nervous System Diseases* / pathology
  • Nervous System Diseases* / physiopathology
  • Nervous System Diseases* / therapy
  • Neurodegenerative Diseases* / pathology
  • Neurodegenerative Diseases* / physiopathology
  • Neurodegenerative Diseases* / therapy
  • Transduction, Genetic
  • Trauma, Nervous System* / pathology
  • Trauma, Nervous System* / physiopathology
  • Trauma, Nervous System* / therapy

Substances

  • MicroRNAs