Pharmacological rescue and screening in a Pitt Hopkins model.

PI: Daniel Marenda, PhD

Institution: Drexel University

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Screening drugs in cells and animals is essential to identifying therapeutic compounds and targets for disease. Though screening drugs in cells is fast, these drugs often fail when tested in animal models of a disease. However, screening in animal models (such as mice and rats) is far too expensive and time consuming to make large scale drug screening feasible in most cases. This proposal will utilize a novel animal model for Pitt Hopkins by using the fruit fly Drosophila melanogaster to screen for potential therapeutic compounds for the treatment of this disorder. Drosophila offer a powerful, well tested, cheap, and quick way to test for new drugs in an established animal model.

Using an established Drosophila model for Pitt Hopkins, we performed a small drug screen and identified three drugs that have been previously identified as having potential for therapeutic use in Pitt Hopkins, suggesting that this model is capable of identifying molecules with the potential for further exploration. We are expanding upon this approach, and will use this animal model to screen for additional compounds in a large library of drugs. Our hope is to identify new compounds in this animal model which can be further explored in mammalian models of Pitt Hopkins, and eventually be furthered explored in humans.