Clinical status of duplex RNA

Abstract

Double-stranded RNA has become a ubiquitous tool for inhibition of gene expression in the laboratory. If similar success could be achieved in vivo, duplex RNA might provide a new class of therapeutics capable of treating a broad spectrum of disease. Chemists and biologists developing duplex RNA as a drug have made progress but continue to face challenges. This review presents the current status of duplex RNA in the clinic and comments on future prospects for the approach.

Figure 1

(a) Structure of a typical siRNA. (b) Publications citing “siRNA” from 2001 to 2009.

Figure 2

Local and systemic siRNA drugs in clinical trials by year.

Figure 3

siRNA drugs in clinical trials classified by (top to bottom) target tissue, condition, and primary sponsoring company.