Suppression of apoptosis in the liver by systemic and local delivery of small-interfering RNAs

Zender, L., Kubicka, S. (2007) Suppression of apoptosis in the liver by systemic and local delivery of small-interfering RNAs. Methods Mol Biol, 361. pp. 217-26. ISSN 1-59745-208-4

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DOI: 10.1385/1-59745-208-4:217


RNA interference (RNAi) is a sequence-specific gene-silencing mechanism triggered by double-stranded RNA. RNAi was shown to allow transient or stable knockdown of gene expression in a broad range of species and has been used successfully for functional genomic screens in mammalian cells and Caenorhabditis elegans. Standard therapeutic use of RNAi in clinical settings in humans has been hampered by the lack of effective methods to deliver the small-interfering RNAs (siRNAs) or short-hairpin RNA expression vectors into the diseased organs. In mice, systemic delivery of siRNAs by hydrodynamic intravascular injection leads to highly efficient uptake of siRNAs into the liver. Several groups demonstrated therapeutic use of RNAi in mouse models of acute liver failure or hepatitis B virus replication. This chapter will focus on the technical background of hydrodynamic and portal vein delivery techniques in mice and will give practical guidance for using these techniques for siRNA delivery into the liver.

Item Type: Paper
Uncontrolled Keywords: Animals Apoptosis/ drug effects/genetics Caenorhabditis elegans/genetics/metabolism Gene Therapy/methods Genetic Vectors/genetics/metabolism/pharmacology Genomics/methods Hepatitis B/drug therapy/genetics/metabolism Hepatitis B virus/drug effects/genetics/metabolism Injections, Intravenous Liver/ metabolism Liver Failure, Acute/drug therapy/genetics/metabolism Mice RNA Interference RNA, Double-Stranded/genetics/metabolism/ pharmacology RNA, Small Interfering/genetics/metabolism/ pharmacology Virus Replication/drug effects/genetics
Subjects: diseases & disorders
therapies > gene therapy
organism description > animal > mammal > rodent > mouse
organism description > virus
CSHL Authors:
Communities: CSHL labs
Depositing User: CSHL Librarian
Date: 2007
Date Deposited: 30 Aug 2011 18:20
Last Modified: 11 Apr 2018 15:43
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