MicroRNA reexpression as differentiation therapy in cancer

J Clin Invest. 2009 Aug;119(8):2119-23. doi: 10.1172/JCI40107. Epub 2009 Jul 20.


Since their discovery in the early 2000s, microRNAs (miRNAs) and their penchant for RNA interference have taken the scientific community by storm, working their way into virtually every corner of biological inquiry. The very nature of their action, the ability to simultaneously extinguish the expression of a multitude of genes and negate their functions, immediately suggested therapeutic promise. In this issue of the JCI, a step toward the realization of this promise is described. Taulli et al. demonstrate that the miRNAs miR-1/miR-206, which are routinely lost in advanced, poorly differentiated rhabdomyosarcoma (RMS) but characteristically expressed in the mature skeletal muscle from which these tumors arise, restore the myogenic differentiation program and block the tumorigenic phenotype (see the related article beginning on page 2366). Their data support the notion that these small RNAs, effectively functioning as "micro-sheriffs" by restoring myogenic law and order, hold substantial clinical potential as differentiation therapy for RMS and perhaps other solid tumors. miRNA reexpression therapy constitutes a novel approach to handcuff oncogenes and arrest tumor development.

Publication types

  • Comment

MeSH terms

  • Animals
  • Cell Differentiation*
  • Humans
  • Mice
  • MicroRNAs / analysis
  • MicroRNAs / genetics*
  • MicroRNAs / physiology
  • Muscle, Skeletal / cytology
  • Muscle, Skeletal / metabolism
  • Neoplasms / genetics
  • Neoplasms / therapy*
  • Organ Specificity
  • Proto-Oncogene Proteins c-met / physiology
  • Rhabdomyosarcoma / therapy


  • MicroRNAs
  • Proto-Oncogene Proteins c-met