Chemical modification: the key to clinical application of RNA interference?
review · Level V
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- Record sourced from PubMed, PMID 18060019.
- Also identified by PMC identifier 2096450.
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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.
Medical subject headings
- Gene Expression Regulation
- RNA Interference
- RNA Stability
- RNA, Small Interfering