2007 Rustbelt RNA Meeting
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Poster number 69 submitted by Yi Shu

Application of RNA nanotechnology in gene delivery and therapy

Songchuan Guo (Department of Comparative Pathobiology and Department of Biomedical Engineering, Purdue Univeristy), Yi Shu (Department of Biomedical Engineering, University of Cincinnati), Steve Perrin (Department of Biomedical Engineering, University of Cincinnati), Hongkwan Cho (Department of Biomedical Engineering, University of Cincinnati), Jing Liu (Department of Biomedical Engineering, University of Cincinnati), Peixuan Guo (Department of Comparative Pathobiology and Department of Biomedical Engineering, Purdue Univeristy, Department of Biomedical Engineering, University of Cincinnati)

Abstract:
By utilizing RNA nanotechnology, we engineered both therapeutic siRNA and a receptor-binding RNA aptamer into individual pRNAs of phi29's motor. The RNA building block harboring siRNA or other therapeutic molecules was fabricated subsequently into a trimer through the interaction of engineered right and left interlocking RNA loops. The incubation of the protein free nanoscale particles containing the receptor-binding aptamer or other ligands resulted in the binding and co-entry of the trivalent therapeutic particles into cells, subsequently modulating the apoptosis of cancer cells and leukemia model lymphocytes in cell culture and animal trials. The use of such antigenicity-free 20-nm particles holds promise for the repeated long-term treatment of chronic diseases.

Keywords: phi29 motor, pRNA, siRNA