Allele-specific RNA interference for neurological disease

Gene Ther. 2006 Mar;13(6):576-81. doi: 10.1038/


Suppressing the expression of toxic genes through RNAi holds great promise for the treatment of human disease. Allele-specific approaches have now been used to silence dominant toxic genes implicated in several neurological disorders. Here, we review strategies used to achieve allele-specific silencing in light of recent developments in the field of RNAi biology. In particular, new insights into siRNA and miRNA processing may be used to improve efficiency and specificity of RNAi therapy. We further discuss steps that can be taken to maximize the therapeutic benefits of this powerful technology.

Publication types

  • Review

MeSH terms

  • Alleles
  • Animals
  • Forecasting
  • Genes, Dominant
  • Genetic Therapy / methods*
  • Genetic Therapy / trends
  • Humans
  • MicroRNAs / administration & dosage
  • MicroRNAs / genetics
  • Nervous System Diseases / therapy*
  • RNA Interference*
  • RNA, Small Interfering / administration & dosage
  • RNA, Small Interfering / genetics


  • MicroRNAs
  • RNA, Small Interfering