Presenter:
Sophia Garganta
Parkinson's Disease (PD) is a movement disorder characterized primarily by the progressive degeneration of dopaminergic neurons and intraneuronal aggregates composed largely by the α-synuclein protein. Overexpression of SNCA, the gene encoding α-synuclein, is a key genetic factor for PD. In previous in vitro studies, our lab used human induced pluripotent stem cell (hiPSC)-derived dopaminergic neurons and demonstrated that targeted repression of SNCA using an epigenome editing approach significantly reduced α-synuclein levels and rescued disease-related phenotypes. To further elucidate the role of α-synuclein in PD and related diseases, we developed a platform to specifically target the SNCA gene in dopaminergic neurons based on CRISPR/Cas9 technology delivered by an all-in-one adeno-associated viral vector (AAV). The design of the vector leveraged single-cell multi-omic data generated in our lab from the human brain. My project will assess the therapeutic efficacy using a humanized PD-mouse model developed in the lab through immunohistochemical protein assays to measure α-synuclein expression and aggregation in the mouse substantia nigra. Identifying Structural Determinants of Selectivity in Odorant Receptors
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Presenter: Sophia Garganta
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Chemosensation allows mammals to identify food, social cues, and environmental threats, yet the molecular basis by which olfactory receptors recognize specific odor molecules-including how they selectively respond to particular chemical groups-is still not fully understood. Odorant receptors are G protein-coupled receptors responsible for detecting thousands of distinct odor molecules. The aim of this study is to identify odorant receptors that respond to specific odorants and characterize shared sequence features that may be important for odor- driven activation. Activated receptors are identified and analyzed to determine sequence features that may contribute to their activation. The receptors are cloned into a mammalian expression plasmid with modified C-terminus in order to promote cell surface expression and allow for activation in mammalian cells. Receptor activity is quantified using the GloSensor™ cAMP Assay, which enables real-time measurement of GPCR signaling via cAMP levels in living cells. This work supports the validation of sequencing-based data, may help clarify how specific structural elements in receptors drive odor recognition, and could inform future studies of sensory signaling and ligand-receptor specificity.
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Duke University / 2025
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Presenter: Sophia Garganta