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Bioorg Med Chem Lett. 2010 Jun 1;20(11):3203-7. doi: 10.1016/j.bmcl.2010.03.109. Epub 2010 Apr 2.

Clinical status of duplex RNA.

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  • 1Departments of Pharmacology and Biochemistry, UT Southwestern Medical Center at Dallas, 6001 Forest Park Road, Dallas, TX 75390, USA.

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.

Copyright 2010 Elsevier Ltd. All rights reserved.

PMID:
20399650
[PubMed - indexed for MEDLINE]
PMCID:
PMC2874620
Free PMC Article
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