Proof of RNA Interference in Humans after Systemic Delivery of siRNAs

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Abstract

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Great hope is placed in the use of RNA interference in the development of novel therapeutics. Recently, small interfering RNAs (siRNAs) were delivered to tumor cells of melanoma patients by nanoparticles consisting of a cyclodextrin polymer, and polyethylene glycol coupled to adamantane and transferrin (see picture). It was demonstrated that the target mRNA in the patients was degraded by a specific RNA interference mechanism.

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