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.
Original language | English (US) |
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Pages (from-to) | 3615-3622 |
Number of pages | 8 |
Journal | Journal of Clinical Investigation |
Volume | 117 |
Issue number | 12 |
DOIs | |
State | Published - Dec 2007 |
ASJC Scopus subject areas
- Medicine(all)