RNAi therapeutics: a potential new class of pharmaceutical drugs.
The rapid identification of highly specific and potent drug candidates continues to be a substantial challenge with traditional pharmaceutical approaches. Moreover, many targets have proven to be intractable to traditional small-molecule and protein approaches. Therapeutics based on RNA interference (RNAi) offer a powerful method for rapidly identifying specific and potent inhibitors of disease targets from all molecular classes. Numerous proof-of-concept studies in animal models of human disease demonstrate the broad potential application of RNAi therapeutics. The major challenge for successful drug development is identifying delivery strategies that can be translated to the clinic. With advances in this area and the commencement of multiple clinical trials with RNAi therapeutic candidates, a transformation in modern medicine may soon be realized.
Bumcrot
David
D
Alnylam Pharmaceuticals Inc., 300 Third Street, Cambridge, MA 02142, USA.
Manoharan
Muthiah
M
Koteliansky
Victor
V
Sah
Dinah W Y
DW
D000818:Animals; D002986:Clinical Trials as Topic; D004195:Disease Models, Animal; D015195:Drug Design; D006801:Humans; D008268:Macular Degeneration / Q000188:drug therapy; D034622:RNA Interference; D018357:Respiratory Syncytial Virus Infections / Q000188:drug therapy