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

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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 languageEnglish (US)
Pages (from-to)3615-3622
Number of pages8
JournalJournal of Clinical Investigation
Issue number12
Publication statusPublished - Dec 2007


ASJC Scopus subject areas

  • Medicine(all)

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