Doctor of Philosophy (PHD)
Biochemistry and Molecular Biology (Medicine)
Date of Defense
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Despite significant resource expenditures and decades of focused research, chronic age-associated diseases have not been met with improvements in patient outcomes that reflect the successes against infectious disease over the last century. This observation calls into question contention approaches to drug discovery for diseases of aging, and more importantly, their molecular targets. Herein, the strategies for engineered negligible senescence (SENS) framework for drug discovery -- an approach to treating aging broadly based on damage mitigation -- is presented. A translational program for age-related macular degeneration is detailed as illustration of this approach. Collectively, this work validates the academic and commercial viability of the SENS damage repair platform and presents a novel therapeutic modality that may eventually treat the global leading cause of vision loss in the elderly.
macular degeneration; sens; Stargardt; enzyme replacement therapy
Moody, Kelsey James, "A Translational Approach to Enzyme-Mediated Retinal Lipofuscin Removal for Atrophic Age-Related Macular Degeneration and Stargardt's Disease" (2019). Open Access Dissertations. 2261.