Journal
JOURNAL OF CLINICAL INVESTIGATION
Volume 117, Issue 12, Pages 3615-3622Publisher
AMER SOC CLINICAL INVESTIGATION INC
DOI: 10.1172/JCI33483
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Funding
- NIGMS NIH HHS [R01 GM073042] Funding Source: Medline
- PHS HHS [73042, 60642] Funding Source: Medline
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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.
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