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Mentor: Kevin G. Rice, PhD
Year Entered Into Program: 2004-2009
PhD Institution: University of Iowa, 2009
Gene therapy has not reached its full potential in the prevention and treatment of disease. Non-viral vectors are gaining wide utility as a means of gene delivery due to the control one has when synthesizing a carrier, which can lead to increases in efficacy and safety. Non-viral vectors can be constructed efficiently and with high reproducibility. In addition, non-viral DNA condensates can be dosed repeatedly with little or no immune response or cytotoxicity. Two important elements in designing a non-viral vector are to design a compound which targets a specific receptor or cell type, and to devise a means of protecting therapeutic DNA from intracellular degradation and DNAses. The focus of my research in the Rice lab is to synthesize DNA carrier molecules with a ligand specific to the mannose receptor, and with properties which render it resistant to intracellular degradation. Because our DNA condensates are mannoseylated, we have been able to target Kupffer cells in-vivo with high specificity. In our future work we will be targeting Dendritic cells bearing the SIGN receptor, a related C-type lectin which binds mannose. DC-SIGN mediates transient adhesion with T-cells, and this contact is essential in initiating a primary immune response.
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