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Article
Expanding RNAi therapeutics to extrahepatic tissues with lipophilic conjugates
Therapeutics based on short interfering RNAs (siRNAs) delivered to hepatocytes have been approved, but new delivery solutions are needed to target additional organs. Here we show that conjugation of 2′-O-hexadecy...
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Article
The measurement of CSF flow through the aqueduct in normal and hydrocephalic children: from where does it come, to where does it go?
Despite 100 years of study, the theories of cerebrospinal fluid (CSF) formation and absorption remain controversial. Measuring CSF flow through the aqueduct using magnetic resonance imaging (MRI) provides a un...
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Article
Target accessibility dictates the potency of human RISC
In this report, we examined the effect of increased target site access on activated human RNA-induced silencing complex (RISC*) catalysis. Kinetic studies revealed that siRNA-programmed RISC* cleaved target RNA w...
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Article
Specific and potent RNAi in the nucleus of human cells
RNA interference (RNAi) has become a research tool to control gene expression in various organisms and holds potential as a new therapeutic strategy. The mechanism of small interfering RNA (siRNA)-mediated RNA...