Current prospects for RNA interference-based therapies

Abstract

RNA interference (RNAi) is a powerful approach for reducing expression of endogenously expressed proteins for biological applications, or targeting the expression of pathological proteins for therapy. Several delivery methods are available to achieve RNAi in ex vivo and in vivo settings for therapeutic results. The development of RNAi-based therapeutics has advanced sufficiently to allow human clinical trials to begin. Here we outline the broad range of cell-, tissue- and disease-specific approaches under investigation for RNAi therapeutics. The barriers posed by certain cells and tissues are described, as are issues with off-target silencing.