Chemical modification: the key to clinical application of RNA interference?
Open Access
- 3 December 2007
- journal article
- review article
- Published by American Society for Clinical Investigation in Journal of Clinical Investigation
- Vol. 117 (12) , 3615-3622
- https://doi.org/10.1172/jci33483
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.Keywords
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