Development of a Two-Vector Approach to Cell Specific In Utero Gene Therapy
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Authors
Tschernia, Nicholas
Issue Date
2010
Type
Thesis
Language
en_US
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Abstract
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.
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In Copyright(All Rights Reserved)