Using RNAi in the clinic.
RNA interference (RNAi) is a biological process that controls gene silencing in all living cells. Targeting the RNAi system represents a novel therapeutic strategy that has the ability to intercede with multiple disease-related genes and so target many human diseases. Recently, the design of small interfering RNA (siRNA)-selective compounds has become more straightforward because of the significant progress made in predictive modeling. Exciting new data show that siRNA blocks gene function in vivo, suggesting that it has potential as a therapeutic approach for humans. This article highlights recent advances in siRNA research and discusses the aims of the pharmaceutical and biotechnology industries to progress siRNA compounds into the clinical. In the fight against disease, the use of RNAi is a powerful new tactic that adds to the arsenal of small-molecule inhibitors and antibody-blocking approaches.
Dev
Kumlesh K
KK
Novartis Institutes for BioMedical Research, Neuroscience Research, Novartis Pharma AG, WSJ 386-7-43, CH-4002 Basel, Switzerland. kumlesh_k.dev@novartis.com
D000818:Animals; D002986:Clinical Trials as Topic; D003550:Cystic Fibrosis / Q000628:therapy; D016503:Drug Delivery Systems / Q000379:methods; D015195:Drug Design; D004345:Drug Industry / Q000379:methods; D015870:Gene Expression / Q000187:drug effects / Q000235:genetics; D006801:Humans; D008954:Models, Biological; D019636:Neurodegenerative Diseases / Q000628:therapy; D034622:RNA Interference; D034741:RNA, Small Interfering / Q000235:genetics*; D042461:Vascular Endothelial Growth Factor A / Q000037:antagonists & inhibitors / Q000235:genetics; D014764:Viral Proteins / Q000037:antagonists & inhibitors / Q000235:genetics; D014777:Virus Diseases / Q000628:therapy
D034741:RNA, Small Interfering; D042461:Vascular Endothelial Growth Factor A; D014764:Viral Proteins