MicroRNAs: novel therapeutic targets in neurodegenerative diseases

Drug Discov Today. 2009 Dec;14(23-24):1123-9. doi: 10.1016/j.drudis.2009.09.009. Epub 2009 Oct 12.


The prevalence of neurodegenerative disorders is rising steadily as human life expectancy increases. However, limited knowledge of the molecular basis of disease pathogenesis is a major hurdle in the identification of drug targets and development of therapeutic strategies for these largely incurable disorders. Recently, differential expression of endogenous regulatory small RNAs, known as 'microRNAs' (miRNAs), in patients of Alzheimer's disease, Parkinson's disease and models of ataxia suggest that they might have key regulatory roles in neurodegeneration. miRNAs that can target known mediators of neurodegeneration offer potential therapeutic targets. Our bioinformatic analysis suggests novel miRNA-target interactions that could potentially influence neurodegeneration. The recent development of molecules that alter miRNA expression promises valuable tools that will enhance the therapeutic potential of miRNAs.

Publication types

  • Review

MeSH terms

  • Animals
  • Brain / metabolism*
  • Genetic Predisposition to Disease
  • Humans
  • MicroRNAs / physiology*
  • Neurodegenerative Diseases / genetics*
  • Neurodegenerative Diseases / therapy


  • MicroRNAs