Mechanisms of inherited neurodegenerative diseases

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The majority of our progress made during the past year has been in setting up our new lab. We purchased necessary equipment and reagents to perform stem cell culture and neuron differentiation. We setup an automated microscope to facilitate data collection related to lysosomal dysfunction in stem cell-derived neurons. Using this microscope, we have begun to carefully characterize the lysosomal phenotype of our cellular model of progranulin-related FTD. In parallel, we have begun to optimize new proteomic approaches to determine how lysosomes change in protein composition in neurons that lack progranulin, in collaboration with the NINDS proteomics facility. During the past fiscal year, we have recruited a post-bac research assistant (Rajan Patel) and a lab manager (Maia Parsadanian). During the last quarter, we recruited two MD/PhD students (Michael Fernandopulle and Stewart Humble).