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J Clin Invest. 2007 Dec;117(12):3615-22.

Chemical modification: the key to clinical application of RNA interference?

Author information

1
Department of Pharmacology, University of Texas Southwestern Medical Center, Dallas, Texas 75390-9041, USA. david.corey@utsouthwestern.edu

Abstract

RNA interference provides a potent and specific method for controlling gene expression in human cells. To translate this potential into a broad new family of therapeutics, it is necessary to optimize the efficacy of the RNA-based drugs. As discussed in this Review, it might be possible to achieve this optimization using chemical modifications that improve their in vivo stability, cellular delivery, biodistribution, pharmacokinetics, potency, and specificity.

PMID:
18060019
PMCID:
PMC2096450
DOI:
10.1172/JCI33483
[Indexed for MEDLINE]
Free PMC Article

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