RNA interference: from basic research to therapeutic applications.
An efficient mechanism for the sequence-specific inhibition of gene expression is RNA interference. In this process, double-stranded RNA molecules induce cleavage of a selected target RNA (see picture). This technique has in recent years developed into a standard method of molecular biology. Successful applications in animal models have already led to the initiation of RNAi-based clinical trials as a new therapeutic option.Only ten years ago Andrew Fire and Craig Mello were able to show that double-stranded RNA molecules could inhibit the expression of homologous genes in eukaryotes. This process, termed RNA interference, has developed into a standard method of molecular biology. This Review provides an overview of the molecular processes involved, with a particular focus on the posttranscriptional inhibition of gene expression in mammalian cells, the possible applications in research, and the results of the first clinical studies.
Kurreck
Jens
J
Institute of Industrial Genetics, University of Stuttgart, Allmandring 31, 70569 Stuttgart, Germany. jens.kurreck@iig.uni-stuttgart.de
D000818:Animals; D016503:Drug Delivery Systems; D005786:Gene Expression Regulation / Q000187:drug effects; D006801:Humans; D035683:MicroRNAs / Q000235:genetics / Q000378:metabolism; D009369:Neoplasms / Q000235:genetics / Q000628:therapy; D034622:RNA Interference; D012330:RNA, Double-Stranded / Q000378:metabolism; D034741:RNA, Small Interfering / Q000737:chemistry*; D014777:Virus Diseases / Q000628:therapy
D035683:MicroRNAs; D012330:RNA, Double-Stranded; D034741:RNA, Small Interfering