Molecular Biology: Clinical Promise for RNA Interference

RNA interference, a naturally occurring form of gene regulation, can be induced experimentally by treatment with small interfering RNAs (siRNAs), double-stranded RNA molecules designed to interfere specifically with key genes. In this study, biopsies from three melanoma patients who had received siRNA infusions revealed dose-dependent reductions in the levels of target messenger RNA and protein.

The results form part of the first in-human phase I clinical trial of systemic siRNA treatment for patients with solid cancers using a targeted, nanoparticle delivery system -- designed to help the siRNA get to where it needs to go. Mark E. Davis and colleagues show that the injected siRNA yields its effects by RNAi, a feat hitherto demonstrated in mouse and non-human primate models. Because RNAi is likely to be applicable to any gene, the study hints that targeted delivery of siRNA by nanoparticles may prove a useful way of controlling many previously unreachable drug targets.

Author contact:

Mark E. Davis (California Institute of Technology, Pasadena, CA, USA)
E-mail: mdavis@cheme.caltech.edu

Abstract available online.

(C) Nature press release.

Message posted by: Trevor M. D'Souza