Mitochondrical Gene Therapy
Khan, Shaharyar M., Department of Medicine, University of Virginia
Bayliss, Douglas, Department of Medicine, University of Virginia
Lee, Kevin, Department of Neuroscience, University of Virginia
Many diseases arise from mutations in mitochondrial DNA (mtDNA). These conditions typically affect non-mitotic tissues (brain, retina, muscle) and are largely untreatable. Evidence is accumulating for mtDNA abnormalities in diseases such as Alzheimer's, Parkinson's and type II diabetes. Research and treatment of these conditions is hampered by lack of methods for manipulation of the mitochondrial genome in situ. This dissertation presents the development of technologies and the insights they reveal for delivery of the entire human mitochondrial genome into cells and animals opening the way for the treatment of diseases arising from defects in mitochondrial DNA.
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PHD (Doctor of Philosophy)
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