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An Evolutionary Approach to Vulnerability Mapping in Order to Identify Alternative and Synergistic Therapeutic Strategies for TSC and Related Diseases

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[Technical Report, Final Report]

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The aim of this research project is to develop new approaches to the treatment of diseases resulting from mutations in the Tuberous sclerosis complex TSC genes. TSC mutations lead to the formation of tumors in tissues including the brain, skin, kidneys, heart and lungs and affect an estimated 1 in 6,000 to 10,000 births. Furthermore, disruption of TSC can produce varied neurological and cognitive deficits, representing the most severe features of TSC. The currently available approaches to treating TSC-related diseases are limited and generally block or slow down tumor growth, rather than killing the diseased cells. Therefore, there is an urgent need to develop new therapeutic strategies to treat TSC related diseases. The purpose of our work is to identify new drug targets that selectively kill TSC cells either alone or in combination with RapamycinRapalogs that are used today for the treatment of TSC. Rapalogs have shown some success in treating TSC tumors but their effects are cytostatic and tumors rapidly regrow upon cessation of treatment, highlighting the urgent need to identify new drugs for the treatment of TSC. To achieve this goal, we will use state-of-the art functional genomics methods in the fruit fly, Drosophila, a proven model to study TSC, to identify drug targets that synergize with Rapalogs in the treatment of TSC. In addition, we will characterize in details a promising drug target that has already emerged from our screens for the treatment of TSC.

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  • Medicine and Medical Research

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[A, Approved For Public Release]