Targeting neurological disease with RNAi.
The neuroscientific community rapidly adopted RNA interference techniques as an experimental tool for the dissection of gene function in vitro and in animal models of neurological disease in vivo. Here, we discuss recent advances in the biotechnical implementation of siRNA/shRNA-mediated gene silencing focusing on issues of design, delivery and putative detrimental effects. We then summarize the current use of RNAi in targeting neurological disease models and give an outlook on the implementation of this technique in clinical therapy.
Lingor
Paul
P
Dept. of Neurology, Georg-August-University Göttingen, Faculty of Medicine, Waldweg 33, 37073 Göttingen, Germany. plingor@gwdg.de
Bähr
Mathias
M
D001921:Brain / Q000378:metabolism / Q000473:pathology; D006801:Humans; D009422:Nervous System Diseases / Q000235:genetics / Q000628:therapy*; D034622:RNA Interference; D034741:RNA, Small Interfering / Q000235:genetics / Q000378:metabolism; D014162:Transfection
D034741:RNA, Small Interfering