RNAi in drug development: frequently asked questions

摘要

The RNA interference phenomenon (RNAi) was first described in the early 1990's, but was not quite understood until Andrew Fire and Craig Mello described their work with C. elegans in 1998 [1]. The subsequent demonstration that RNAi can also occur in mammalian cells [2] triggered tremendous interest towards this phenomenon, particularly for functional genomics applications. RNAi offers a powerful tool for ascribing functions to genes, while the application of RNAi technology to in vivo models of disease opens up tremendous opportunities to develop a novel generation of oligonucleotide-based drugs.