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Toxicologic Pathology, Vol. 35, No. 3, 327-336 (2007)
DOI: 10.1080/01926230701197107
© 2007 Society of Toxicologic Pathology

Invited Review

Review of the Application of RNA Interference Technology in the Pharmaceutical Industry

Henny M. Martineau1 and Ian T. Pyrah2

1 Scottish Agricultural College, Allan Watt Building, Bush Estate, Penicuik, EH26 0QE, United Kingdom
2 Amgen Inc, One Amgen Center Drive, Thousand Oaks, CA 91320, USA

Correspondence: Address correspondence to: Ian T. Pyrah, Amgen Inc, One Amgen Center Drive, Mailstop 29-2-A, Thousand Oaks, CA, 91320, USA; e-mail:ipyrah{at}amgen.com

Ribonucleic acid (RNA) interference (RNAi) is a recently discovered phenomenon whereby the introduction of double stranded (ds) RNA into the cytoplasm of the cell results in the specific and efficient degradation of complementary messenger (m) RNA and, therefore, reduced protein production. It was discovered by chance during attempts to develop flowers with increased colour intensity. The specific nature of the inhibition of protein production of cells has resulted in an explosion of research to understand and exploit RNAi. The technique is now established in in vitro systems, and much work is focussed in adapting RNAi for in vivo application. The potential of the technology in understanding physiological and pathological processes is significant, while its development as a therapeutic agent holds much promise as targeted agents. This review will describe the basic biological processes that drive RNAi, indicate current areas of areas research, and forecast future areas of development.

Key Words: RNA interference • biotechnology • discovery pathology • drug development • mechanisms of toxicity • molecular pathology

Abbreviations: ATP, adenosine triphosphate • CHS, chalcone synthetase • CIA, collagen induced arthritis • CL, cationic liposomes • ds, double stranded • mi, micro • nt, nucleotide • piRNA, piwi interacting RNA • PTGS, post transcriptional gene silencing • RdRp, RNA dependent RNA polymerases • RNA, ribonucleic acid • RNAi, RNA interference • RISC, RNA interfering silencing complex • sh, short hairpin • si, short interfering • siRNA, short interfering RNA • sm, small modulatory


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