[RNA interference--towards individualized genetic medicine].
RNA interference (RNAi) is an evolutionarily conserved cellular mechanism by which gene expression is suppressed by double-stranded RNA (dsRNA) in a sequence-specific manner. Enzymatically processed dsRNA molecules (siRNAs) target and facilitate cleavage of mRNA. By harnessing the RNAi machinery using synthetic or DNA-encoded RNA effectors, the siRNA technology offers new therapeutic strategies for genetic diseases. We outline the basic mechanisms of RNAi and present state-of-the-art examples of in vivo siRNA delivery towards development of RNAi-based individualized drugs.
Corydon
Thomas Juhl
TJ
Aarhus Universitet, Institut for Human Genetik, Arhus C.
Mikkelsen
Jacob Giehm
JG
D001709:Biotechnology; D002986:Clinical Trials as Topic; D004345:Drug Industry; D015316:Genetic Therapy / Q000639:trends; D006801:Humans; D034622:RNA Interference; D012330:RNA, Double-Stranded / Q000235:genetics; D034741:RNA, Small Interfering / Q000235:genetics
D012330:RNA, Double-Stranded; D034741:RNA, Small Interfering