differentiation, Genetic Repair and Reporter

分化、基因修复和报告基因

• 批准号: 8295406
• 负责人: LORENZ P. STUDER
• 金额: $ 18.41万
• 依托单位: MCLEAN HOSPITAL
• 依托单位国家: 美国
• 财政年份: 2012
• 资助国家: 美国
• 起止时间: 2012-07-01 至 2014-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8295406
• 关键词: Biological Assay; Budgets; Cell Differentiation process; Cell Line; Cell physiology; Cells; Collaborations; Deposition; Disease model; Foundations; Functional disorder; Genetic; Genetic Drift; Genomics; Human; Institutes; LRRK2 gene; Laboratories; Midbrain structure; Neurons; New York; Patients; Protocols documentation; Psychological Transfer; Reporter; Resources; Stem cells; Technology; Training; Translational Research; Work; biobank; dopaminergic neuron; drug discovery; induced pluripotent stem cell; member; novel strategies; repaired

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.

分化，遗传修复和报告核心将为新兴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编程CRO（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，将高质量的患者特异性iPS细胞系转化为检测方法，以检查源自遗传形式PD患者的腹侧中脑（VM）多巴胺能（DA）神经元的脆弱性，用于转化研究和药物发现。PDiPS联盟计划在第一年将更多的资源和预算用于该核心，以加快为整个联盟提供新方案、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在运送到实验室之前将细胞分化为神经元。