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Development of a Two-Vector Approach to Cell Specific In Utero Gene Therapy
Biochemistry and Molecular Biology
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Genes are vital to every organism; they preserve all of the information that directs and controls the myriad of intricate processes required for both the development and self-sustaining capacity of an organism. Attesting to the need for perfect fidelity, over 10,000 diseases are currently known that are caused by mutations in only a single gene, the so-called “monogenic diseases.” Of course, countless more complex diseases are also caused by alteration within multiple genes. Prior research has proven the feasibility of performing gene therapy, that is, delivering normal healthy copies of genes to alleviate symptoms and treat the organism. However, efficacy and safety must be improved to realize gene therapy’s full potential. To help move toward the full potential of gene therapy, my project utilized a highly cell specific, two-vector delivery system to target a corrective copy of the Factor 8 (FVIII) gene to the cell type hypothesized to be normally responsible for synthesizing this clotting factor (hepatocytes), and thereby correct hemophilia A in a unique sheep model system our laboratory recently re-established.