Therapeutic siRNAs.
The ability of small-interfering RNAs (siRNAs) to silence gene expression in somatic mammalian cells has provided researchers with a novel tool to block the expression of disease-causing genes, provided that their mRNA sequences are known. siRNAs can be delivered to cells either exogenously as synthetic agents or endogenously as gene-encoding siRNAs. Recent studies demonstrate the general application of siRNAs to silence gene expression in a range of cell types and in whole mammals. Beyond their value for dissecting gene functions and target validation, siRNAs also hold great potential as gene-specific therapeutic agents.
Sioud
Mouldy
M
The Norwegian Radium Hospital, Department of Immunology, Molecular Medicine Group, Montebello, N-310, Oslo, Norway. mosioud@ulrik.uio.no
D000818:Animals; D002478:Cells, Cultured; D004195:Disease Models, Animal; D015972:Gene Expression Regulation, Neoplastic / Q000187:drug effects*; D006801:Humans; D008967:Molecular Biology; D034741:RNA, Small Interfering / Q000008:administration & dosage / Q000235:genetics / Q000627:therapeutic use*; D014777:Virus Diseases / Q000235:genetics / Q000628:therapy*
D034741:RNA, Small Interfering