Mechanisms of inherited neurodegenerative diseases

在过去的一年里，我们取得的大部分进展都是建立我们的新实验室。我们购买了必要的设备和试剂来进行干细胞培养和神经元分化。我们建立了一个自动化显微镜，以促进与干细胞衍生的神经元中溶酶体功能障碍相关的数据收集。使用该显微镜，我们开始仔细表征前颗粒蛋白相关FTD细胞模型的溶酶体表型。与此同时，我们已经开始优化新的蛋白质组学方法，以确定缺乏颗粒蛋白前体的神经元中溶酶体蛋白质组成的变化，与NINDS蛋白质组学设施合作。在过去的一个财政年度，我们已经招募了一个后bac研究助理（拉詹帕特尔）和实验室经理（马亚Parsadanian）。在上个季度，我们招募了两名MD/PhD学生（Michael Fernandopulle和Stewart Humble）。

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).