Potent and persistent in vivo anti-HBV activity of chemically modified siRNAs

Nat Biotechnol. 2005 Aug;23(8):1002-7. doi: 10.1038/nbt1122. Epub 2005 Jul 24.

Abstract

The efficacy of lipid-encapsulated, chemically modified short interfering RNA (siRNA) targeted to hepatitis B virus (HBV) was examined in an in vivo mouse model of HBV replication. Stabilized siRNA targeted to the HBV RNA was incorporated into a specialized liposome to form a stable nucleic-acid-lipid particle (SNALP) and administered by intravenous injection into mice carrying replicating HBV. The improved efficacy of siRNA-SNALP compared to unformulated siRNA correlates with a longer half-life in plasma and liver. Three daily intravenous injections of 3 mg/kg/day reduced serum HBV DNA >1.0 log(10). The reduction in HBV DNA was specific, dose-dependent and lasted for up to 7 d after dosing. Furthermore, reductions were seen in serum HBV DNA for up to 6 weeks with weekly dosing. The advances demonstrated here, including persistence of in vivo activity, use of lower doses and reduced dosing frequency are important steps in making siRNA a clinically viable therapeutic approach.

MeSH terms

  • Animals
  • Antiviral Agents / administration & dosage
  • Carcinoma, Hepatocellular / genetics
  • Carcinoma, Hepatocellular / metabolism
  • Carcinoma, Hepatocellular / virology
  • Cell Line, Tumor
  • Coated Materials, Biocompatible / administration & dosage
  • Coated Materials, Biocompatible / chemistry
  • Drug Delivery Systems / methods*
  • Female
  • Gene Targeting / methods
  • Genetic Therapy / methods
  • Hepatitis B / genetics
  • Hepatitis B / metabolism
  • Hepatitis B / therapy*
  • Hepatitis B / virology*
  • Hepatitis B virus / drug effects
  • Hepatitis B virus / genetics*
  • Humans
  • Liposomes / chemistry*
  • Liposomes / pharmacokinetics
  • Liver / drug effects
  • Liver / metabolism
  • Male
  • Metabolic Clearance Rate
  • Mice
  • RNA, Small Interfering / administration & dosage*
  • RNA, Small Interfering / chemistry*
  • RNA, Small Interfering / genetics
  • RNA, Small Interfering / pharmacokinetics
  • Tissue Distribution
  • Treatment Outcome

Substances

  • Antiviral Agents
  • Coated Materials, Biocompatible
  • Liposomes
  • RNA, Small Interfering