Chemical modification: the key to clinical application of RNA interference?
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.
Department of Pharmacology, University of Texas Southwestern Medical Center, Dallas, Texas 75390-9041, USA. david.corey@utsouthwestern.edu
David R
DR
Corey
D005786:Gene Expression Regulation / Q000187:drug effects*; D006801:Humans; D034622:RNA Interference / Q000187:drug effects*; D020871:RNA Stability; D034741:RNA, Small Interfering / Q000737:chemistry / Q000494:pharmacology / Q000627:therapeutic use
D034741:RNA, Small Interfering