Current prospects for RNA interference-based therapies

摘要: RNA interference (RNAi) is a powerful approach for reducing expression of endogenously expressed proteins. It widely used biological applications and being harnessed to silence mRNAs encoding pathogenic proteins therapy. Various methods — including delivering oligonucleotides expressing RNAi triggers from viral vectors have been developed successful in cell culture vivo. Recently, RNAi-based gene silencing approaches demonstrated humans, ongoing clinical trials hold promise treating fatal disorders or providing alternatives traditional small molecule therapies. Here we describe the broad range achieve targeted therapy, discuss important considerations when developing use review current status trials.