US – Page 269 – TechConnect Briefs

Utility of Customized Targeted PEG Linkers for the delivery of oligonucleotides without the use of transfection reagents

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

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 [...]

Development of Peptide Nucleic Acid Assemblies for Application in Drug Delivery and Molecular Sensing

Butler F.N., Elibol O., Hines B.D., Reddy Jr. B., Bashir R., Bergstrom D.E., Purdue University, US

We are exploring peptide nucleic acid (PNA) assemblies as interesting scaffolds for the fabrication of nanomaterials relevant to the diagnosis and treatment of disease. The first assembly is a PNA/LNA (locked nucleic acid) duplex employed [...]

Gene Delivery and Transfection Studies with Lipopolyplexes in Human Endothelial and Smooth Muscle Cells

Brito L., Little S., Langer R., Amiji M., Northeastern University, US

Non-viral gene delivery and transfection in primary human vascular endothelial cells (EC) and smooth muscle cells (SMC) has tremendous potential for cardiovascular diseases, such as in treatment of coronary restenosis. Using a combination of synthetic [...]

Cell-mediated, heat-based therapy for combating HIV infection

Denver H.T., de Noronha C.M., Borca-Tasciuc D-A., Rensselaer Polytechnic Institute, US

There is an increased interest in developing alternative therapies to combat deadly infectious disease including HIV. This work investigates the fundamentals of a novel, nanoparticle-based potential treatment method employing macrophage cells. Macrophages are a very [...]

SiRNA for Therapeutic Immuno-modulation: Simultaneous Delivery of Cytokine-targeted SiRNA and DNA Antigens to Dendritic Cells using Polymer Microcarriers

Singh A., Nie H., Ghosn B., Roy K., The University of Texas at Austin, US

RNA interference mediated gene knockdown is a potential immuno-modulatory tool for tuning dendritic cells (DC) activation and function in-vivo. We have developed a novel multi-tiered delivery system for combinatorial administration of SiRNA and plasmid DNA [...]

Toward in vivo Targeting Delivery of siRNA for Efficient Cancer Therapy

Taratula O., Kirkpatrick P., Salva R., Pandya I., Minko T., He H., Rutgers-Newark University, US

There is an increasing enthusiasm for developing therapies based on RNA interference (RNAi). However, the main obstacle in siRNA therapy is RNA delivering to the cytoplasm where it can guide sequence-specific mRNA degradation. An efficient, [...]

Labile Catalytic Packaging and Delivering of Short Interference RNA to Cancer Cells: Control of Gold Nanoparticles “out” of RNA Complexes

Chen A.M., Taratula O., Wei D., Minko T., He H., Rutgers, The State of New Jersey, US

We report a novel approach to efficiently package and deliver siRNAs with low generation dendrimers by using Au nanoparticles as a “labile catalytic” packaging agent. The Au nanoparticles helped low generation dendrimers to package siRNA [...]

Efficient siRNA Delivery by Secondary and Tertiary Amine Modified Polysaccharides

Ghosn B., Singh A., Roy K., The University of Texas at Austin, US

As siRNA mediated therapy further develops, the need for biocompatible, effective delivery vectors, especially for mucosal delivery, is apparent. We report here a novel nanocarrier for siRNA delivery via targeted chemical modification of the polysaccharide [...]

Systemic Delivery of Targeted, siRNA-Containing Nanoparticles for the Treatment of Solid Tumors: Concept to Clinic

Heidel J.D., Liu J.Y-C., Zeidan R.K., Liang Y., Rele S., Davis M.E., Calando Pharmaceuticals, US

Calando Pharmaceuticals is developing siRNA-containing nanoparticle formulations, and its leading formulation that will enter the clinic in 2008 contains the human transferrin protein as a targeting ligand and siRNA targeting the M2 subunit of ribonucleotide [...]

Oral Delivery of Map4K4 siRNA to Macrophages Inhibits TNF alpha Production and Protects against a LPS-Galactosamine Challenge in Mice

Aouadi M., Tesz G., Soto E., Wang M., Nicoloro S., Ostroff G., Czech M., University of Massachusetts Medical School, US

RNA interference (RNAi) is a new, promising approach to selectively regulate gene expression through modulating mRNA stability and translation. However, the efficient delivery of 21 mer dsRNA molecules (siRNA) to affect RNAi remains a challenge. [...]