Date of Defense

Spring 4-20-2007

Department

Biological Sciences

First Advisor

Andre LaRochelle, National Institutes of Health

Second Advisor

John Geiser, Biological Sciences

Third Advisor

David Huffman, Chemistry

Abstract

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).

Access Setting

Honors Thesis-Campus Only

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