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

Research output: Contribution to journalReview article

223 Scopus citations

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 languageEnglish (US)
Pages (from-to)3615-3622
Number of pages8
JournalJournal of Clinical Investigation
Volume117
Issue number12
DOIs
StatePublished - Dec 2007

ASJC Scopus subject areas

  • Medicine(all)

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