Authors: D.W. Bartlett and M.E. Davis
Affilation: California Institute of Technology, United States
Pages: 763 - 766
Keywords: siRNA, transferrin targeting, systemic delivery, bioluminescent imaging
This abstract highlights the results from our latest work in the design and application of targeted nanoparticles for systemic siRNA delivery. Over the past several years, we have performed extensive physicochemical and biological characterization of the cyclodextrin-containing polycation (CDP) and its complexes with a variety of nucleic acid molecules. As a result of these studies, we are now able to estimate the stoichiometry of an individual nanoparticle, including the number of nucleic acid molecules and the number of targeting ligands. We illustrate how this knowledge can be used to correlate the nanoparticle structure with its biological function. Additionally, we will present our latest findings from in vivo studies looking at the ability of these targeted nanoparticles to deliver therapeutic siRNA to tumors after systemic injection in murine cancer models. Data will also be presented to show the effect of siRNA dosing schedule on the subsequent growth of the tumors, as monitored by bioluminescent imaging.