differentiation, Genetic Repair and Reporter

分化、遗传修复和报告核心将为一种新兴的疾病建模技术引入定向IPS细胞分化、人类基因组编辑和细胞纯化的基本新方法。 分化、遗传修复和报告核心与重新编程CROS(哈佛干细胞研究所和纽约干细胞基金会)以及细胞功能和病理生理学核心紧密结合，将高质量的患者特定iPS细胞系转化为检测腹侧中脑(VM)多巴胺(DA)能神经元的脆弱性，这些神经元来自遗传型帕金森病患者，用于翻译研究和药物研发。PDPS联盟计划在第一年为这一核心使用更多的资源和预算，以加快为整个联盟提供新的方案、培训和基因修复的iPS细胞的工作。在第二年，重点将放在实验室的合作和科学需求上，这些实验室可能需要将分化后的细胞转化为神经元，然后再运往实验室。

The Differentiation, Genetic Repair and Reporter Core will introduce essential new approaches to directed IPS cell differentiation, human genomic editing and cell purification for an emerging disease modeling technology. The Differentiation, Genetic Repair and Reporter Core is closely integrated with the Reprogramming CROs (Harvard Stem Cell Institute and New York Stem Cell Foundation) and the Cell Function and Pathophysiology Core to transition high quality patient-specific iPS cell lines into assays to examine the vulnerability of ventral midbrain (VM) dopaminergic (DA) neurons derived from patients with genetic forms of PD for translational research and drug discovery. The PDiPS Consortium plan is to use more resources and budget for this core in year 1 in order to accelerate the work to provide new protocols, training and genetically repaired iPS cells with reporters for the entire consortium. In year 2, the focus will be on collaboration and scientific needs for laboratories that may require differentiated cells into neurons prior to shipment to their laboratories.