Online Archive of University of Virginia Scholarship
Mitochondrical Gene Therapy903 views
Author
Khan, Shaharyar M., Department of Medicine, University of Virginia
Advisors
Bayliss, Douglas, Department of Medicine, University of Virginia
Lee, Kevin, Department of Neuroscience, University of Virginia
Abstract
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.
Note: Abstract extracted from PDF text
Degree
PHD (Doctor of Philosophy)
Language
English
Rights
All rights reserved (no additional license for public reuse)
Khan, Shaharyar M.. Mitochondrical Gene Therapy. University of Virginia, Department of Medicine, PHD (Doctor of Philosophy), 2006-06-01, https://doi.org/10.18130/V3DF8J.