IDD Models Core

IDD模型核心旨在促进人类干细胞的开发和分析， IDD条件的啮齿动物模型。核心支持分子途径的基础科学研究 以及在影响神经系统的遗传和表观遗传疾病中改变的细胞相互作用。 系统功能此外，该核心还可以进行药物和生物标志物研究，以及临床前研究。 旨在开发IDD的新翻译方法的研究。历史性的力量， IDD模型的核心是使用基于人类多能干细胞（hPSC）的模型， 包括患者特异性诱导多能干细胞（iPSC）和它们通过 基因组编辑，以研究不同类型的IDD条件。核心已纳入小说 通过满足对严格性和再现性的日益增长的需求， 在创建和表征患者特异性 iPSC和hESC。精细的基因编辑能力开发了创新的细胞工具， 神经系统发育和功能改变的复杂分析。这些模型还 提供在疾病相关细胞中评价候选治疗剂的方法，以及 开发新的筛选方法，在小型和大型库中鉴定有前途的化合物 筛选该核心还授权分析IDD条件的啮齿动物模型，不仅通过 为他们的创作提供支持，同时也提供全面和先进的 行为测试服务这项服务包含了一组认知/神经系统 在熟练的核心经理的监督下进行分析，以确保严谨性和可重复性 这些IDD模型研究。我们在下一个项目期间的具体目标是：（1）创造和 分析IDD的人类干细胞模型;和（2）建立和分析IDD的啮齿动物模型。 认识到需要对这些模型进行复杂的分析以阐明发病机制 和新的治疗方法，IDD模型核心使用广泛的现场研究 资源，并利用UW的互补研究核心的扩展网络- 麦迪逊校园提供的基础设施和技术人员，支持集成 分子、基因组、表观基因组、细胞、电生理和行为水平的分析。 集合这些不同的资源旨在扩大IDD模型的范围和深度 该项目旨在促进缺碘症研究从基础科学向转化研究的进展。

The IDD Models Core is designed to foster development and analysis of human stem cell and rodent models of IDD conditions. The core supports basic science studies of molecular pathways and cellular interactions that are altered in genetic and epigenetic disorders affecting nervous system function. In addition, the core enables drug and biomarker studies, as well as preclinical studies that are aimed at developing novel translational approaches to IDD. A historic strength of the IDD Models core is the use of human pluripotent stem cell (hPSC)-based models, which include both patient-specific induced pluripotent stem cells (iPSCs) and their modification by genome editing, to study diverse types of IDD conditions. The core has incorporated novel approaches to meet the increased demands for rigor and reproducibility by meeting international/peer institution standards in the creation and characterization of patient-specific iPSCs and hESCs. The refined gene editing capabilities develop innovative cellular tools for sophisticated analysis of altered nervous system development and function. These models also provide the means to evaluate candidate therapeutics in disorder-relevant cells, as well as develop novel screens to identify promising compounds in small- and large-scale library screening. The core also empowers analysis of rodent models of IDD conditions, not only by providing support for their creation, but also by providing a comprehensive and cutting edge behavioral testing service. The service incorporates a battery of cognitive/nervous system analyses under the supervision of a skilled core manager to ensure the rigor and reproducibility of these IDD model studies. Our specific aims for the next project period are: (1) to create and analyze human stem cell models of IDD; and (2) to create and analyze rodent models of IDD. Recognizing that sophisticated analysis of these models is required to elucidate pathogenesis and novel therapeutic approaches, the IDD Models Core uses extensive on-site research resources and leverages an extended network of complementary research cores on the UW- Madison campus to provide the infrastructure and skilled personnel that support integrated analysis at molecular, genomic, epigenomic, cellular, electrophysiological, and behavioral levels. Assembly of these diverse resources is designed to broaden the scope and depth of IDD model studies and to facilitate progress of IDD research from basic science to translational studies.