Therapeutic short hairpin RNA expression in the liver: viral targets and vectors

Gene Ther. 2006 Mar;13(6):563-75. doi: 10.1038/


Over 500 million people worldwide are infected with one or more different and unrelated types of human hepatitis virus. Such individuals are at a high risk of developing acute or chronic hepatic disease, and ultimately dying from sequelae. Although a vaccine is available for hepatitis A and B virus, treatment options for chronically infected patients are limited, and particularly ineffective in case of hepatitis C virus (HCV) infection. A promising new avenue currently being explored is to harness the power of RNA interference for development of an antiviral therapy. The timing to pursue this particular approach is excellent, with the first in vivo animal models for HCV infection becoming available, and the technology for liver-specific expression of short hairpin RNAs advancing at a rapid pace. Here, we critically review these important current developments, and discuss the next steps to bring this novel approach into the clinics.

Publication types

  • Review

MeSH terms

  • Adenoviridae / genetics
  • Animals
  • Dependovirus / genetics
  • Forecasting
  • Genetic Therapy / methods*
  • Genetic Therapy / trends
  • Genetic Vectors / administration & dosage
  • Genetic Vectors / genetics
  • Hepacivirus / genetics*
  • Hepatitis B virus / genetics
  • Hepatitis C / therapy*
  • Humans
  • Liver / metabolism*
  • RNA Interference*
  • RNA, Small Interfering / metabolism
  • RNA, Small Interfering / therapeutic use*


  • RNA, Small Interfering