Gene therapy strategies for targeting the treatment refractory sites in Hurler syndrome.
2009-03
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Gene therapy strategies for targeting the treatment refractory sites in Hurler syndrome.
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2009-03
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The submitted work details the development of a novel gene therapy vector capable of expressing multiple genes from a single transcript. This vector allows for high level therapeutic gene expression that is coupled to a dual reporter system that allows for real time in vivo tracking of gene expression as well as cellular detection without need for antibody staining. Additionally, a fusion protein was designed to specifically target the α- L -iduronidase protein to the central nervous system by way of the transferrin receptor. This treatment resulted in a decrease in glycosaminoglycan storage material in the brain of mucopolysaccharidosis type I mice. Lastly, we implemented the use of an episomally maintained plasmid-based vector that mediates high levels of protein production in vivo over a long period of time. Cumulatively this work has generated novel findings that will contribute to the field of gene therapy as a whole.
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University of Minnesota Ph.D. dissertation. March 2009. Major: Microbiology, Immunology and Cancer Biology. Advisor: Bruce R. Blazar, M.D. 1 computer file (PDF); viii, 129 pages.
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Osborn, Mark John. (2009). Gene therapy strategies for targeting the treatment refractory sites in Hurler syndrome.. Retrieved from the University Digital Conservancy, https://hdl.handle.net/11299/101635.
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