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Chemical modification: the key to clinical application of RNA interference?
1Department 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.
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