Ribozymes, DNAzymes and small interfering RNAs as therapeutics.
Selective gene silencing by nucleic acid enzymes has provided researchers with a new strategy to block gene expression and drug target validation. Ribozymes, DNAzymes and small interfering RNAs (siRNAs) are being explored as genetic inhibitors of gene expression as well as potential therapeutics against viral infections, inflammatory disorders, haematological diseases and cancers. We review the mode of action of these molecules, with special emphasis on their construction and the possibility to enhance their serum half-lives via specific chemical modifications. Their potential use in cell cultures and in animal models for disease is also highlighted.
Sioud
Mouldy
M
Institute for Cancer Research, Department of Immunology, Molecular Medicine Group, The Norwegian Radium Hospital, Montebello, 0310 Oslo, Norway. mosioud@ulrik.uio.no
Iversen
Per Ole
PO
D000818:Animals; D016207:Cytokines / Q000037:antagonists & inhibitors / Q000235:genetics; D021881:DNA, Catalytic / Q000627:therapeutic use*; D011905:Genes, ras; D006801:Humans; D011493:Protein Kinase C / Q000037:antagonists & inhibitors / Q000235:genetics; D051571:Protein Kinase C-alpha; D016337:RNA, Catalytic / Q000627:therapeutic use*; D034741:RNA, Small Interfering / Q000627:therapeutic use*; D015398:Signal Transduction
D016207:Cytokines; D021881:DNA, Catalytic; D016337:RNA, Catalytic; D034741:RNA, Small Interfering; C496628:PRKCA protein, human; D011493:Protein Kinase C; D051571:Protein Kinase C-alpha