Date of Defense
Andre LaRochelle, National Institutes of Health
John Geiser, Biological Sciences
David Huffman, Chemistry
Gene therapy holds the potential promise to correct disease-causing mutations by delivering therapeutic genes into the genomes of hematopoietic stem cells. Hematopoietic stem cells have been identified as a target for gene therapy due to their unique bility to repopulate and maintain a functional hematopoietic system from the lifetime of an individual. Because hematopoietic stem cells give rise to many different cell types, once a genetically engineered stem cell differentiates, the therapeutic transgene will be found in all the subsequent progency cells. The clinical applications of HSC therapeutic gene therapy have a variety of possible applications. Such genetic blood disorders include aplastic anemia, beta-thalassemia, Blackfan-Diamond syndrome, globoid cell leukodystrophy, sickle-cell anemia, severe combined immunodeficiency (SCID), X-linked lymphoprolifrative syndrome, Wiskott-Aldrich syndrome, and chronic granulomatous disease (CGD).
Clevenger, Joshua R., "Genetic Therapy of Hematopoietic Stem Cells Using a Φ C31 Integrase Based System" (2007). Honors Theses. 150.
Honors Thesis-Campus Only