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Translation of adeno-associate viral vectors for delivery of peptide therapeutics with model-informed dosing
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Burr, Alexandra. Translation of adeno-associate viral vectors for delivery of peptide therapeutics with model-informed dosing. Retrieved from https://doi.org/doi:10.7282/t3-vtcw-8r11

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TitleTranslation of adeno-associate viral vectors for delivery of peptide therapeutics with model-informed dosing
NameBurr, Alexandra (author); Parekkadan, Biju (chair); Cai, Li (member); Roth, Charles (member); Radovick, Sally (member); Rutgers University; School of Graduate Studies
Date Created2022
Other Date2022-01 (degree)
SubjectBiomedical engineering, Gene therapy, Viral vectors
Extent194 pages : illustrations
DescriptionPeptide drugs are a life-saving treatment for millions of patients however, they require repeated administration that impacts efficacy, side effects, and costs which limits their use. An alternative solution to injectable peptide drugs is gene therapy, whereby a patients' cells are genetically modified to encode for a peptide drug leading to more durable treatment that can potentially last for years. Our work focused on developing a mathematical and experimental foundation to translate the use of gene therapeutics for peptide hormones. First, a generalized mathematical model of gene therapeutics that encode a secreted protein was developed for this emerging new class of medicines. Using a newly defined matrix of in vitro bioassays, we verified, for the first time, the accuracy of this model to predict experimental drug concentrations in vivo. Then, using parathyroid hormone (PTH) as proof-of-concept, we designed and tested the bioactivity of a first-in-class PTH gene therapy from AAV vector delivery in cell culture and animal studies. Finally, we used synthetic biology techniques to explore a dynamic way to power the secretion of PTH gene therapy using the body's own circadian rhythms. These results demonstrate feasibility for using AAV gene therapy to deliver protein and hormone therapeutics in a dynamic way.
NotePh.D.
NoteIncludes bibliographical references
Genretheses
Persistent URLhttps://doi.org/doi:10.7282/t3-vtcw-8r11
LanguageEnglish
CollectionSchool of Graduate Studies Electronic Theses and Dissertations
Organization NameRutgers, The State University of New Jersey
RightsThe author owns the copyright to this work.
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