Zhao H., Reddy P., Xia J., Wang M., Sapra P., Bandaru R., Belen Rubio M., Wu D., Zhu P., Filpula D., Greenberger L.M., Horak I.D.
Enzon Pharmaceuticals, Inc., US
Keywords: customized targeted PEG linkers, locked nucleic acid, oligonucleotides
Delivery of LNA oligonucleotides using Customized Targeted PEG linkers has improved the in vitro cellular uptake into cancer cells. Customized Targeted PEG linker-LNA conjugates can knock down target mRNA in a concentration dependent fashion with great sequence specificity. The lead compounds have achieved IC50 values of about 20 nM in A549 human lung cancer cells with no transfection. Positively charged Customized PEG linkers may provide a promising approach for more efficient in vivo delivery of oligonucleotides including LNA and siRNA. Preclinical human xenograft models will be used to assess biodistribution, tumor-specific down-modulation of target mRNA, and efficacy of Customized Targeted PEG Linker technology.
Journal: TechConnect Briefs
Volume: 2, Nanotechnology 2008: Life Sciences, Medicine & Bio Materials – Technical Proceedings of the 2008 NSTI Nanotechnology Conference and Trade Show, Volume 2
Published: June 1, 2008
Pages: 372 - 374
Industry sector: Medical & Biotech
Topics: Biomaterials, Materials for Drug & Gene Delivery
ISBN: 978-1-4200-8504-4