Abstract

The RNA interference (RNAi) pathway specifically silences genes in response to double stranded RNA (dsRNA) triggers that share homology with the targeted gene. Short hairpin RNAs (shRNAs) expressed from transcription templates can act as the dsRNA RNAi trigger. Recently we showed that shRNA expression plasmids could silence luciferase gene expression in adult mice. Furthermore, we showed that shRNA expression plasmids directed against hepatitis B virus (HBV) could reduce the production of HBV RNAs, DNAs and proteins in mice when introduced by hydrodynamic transfection. However, if these promising new therapeutics are to be useful in humans, delivery methods that can safely introduce RNAi triggers into the majority of hepatocytes in humans may be required.

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