¹Departments of Pediatrics and Genetics, School of Medicine, Stanford University, Stanford, CA, USA

Correspondence: Dr MA Kay, Departments of Pediatrics and Genetics, School of Medicine, Stanford University, Room G305, 300 Pasteur Drive, Stanford, CA 94305, USA. E-mail: markay@stanford.edu

Received 14 November 2005; Revised 3 January 2006; Accepted 5 January 2006; Published online 2 February 2006.

Abstract

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.