Mechanisms of inherited neurodegenerative diseases

我们在过去一年中取得的主要进展是建立了我们的新实验室。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。我们设置了一个自动显微镜，以方便收集与干细胞来源的神经元中溶酶体功能障碍相关的数据。使用这种显微镜，我们已经开始仔细表征我们的前颗粒蛋白相关FTD细胞模型的溶酶体表型。同时，我们已经开始优化新的蛋白质组学方法，以确定缺乏颗粒前蛋白的神经元中溶酶体如何改变蛋白质组成，与NINDS蛋白质组学设施合作。在过去的财政年度，我们已经招募了一名博士后研究助理（Rajan Patel）和一名实验室经理（Maia Parsadanian）。在上个季度，我们招募了两名博士研究生（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).