Preclinical Models: Core F

（临床前模型核心（PMC）：核心F） 项目摘要 描述：临床前模型核心（PMC）支持IDDRC用户寻求新的IDD治疗方法， 在3种临床前模型中研究这些疾病：（1）啮齿动物模型： 表征遗传或后天残疾的小鼠模型的行为。测量的行为包括 学习、记忆、社会偏好、超声波发声、睡眠和焦虑。(2)干细胞模型：这 核心组件通过生成诱导多能干细胞（iPSC）帮助用户创建疾病模型 使用标准的重编程技术或通过基因组编辑，如CRISPR-Cas， 线这些细胞，有些来自IDD患者，提供了一个模型，在其中仔细检查细胞 遗传疾病的后果，并评估可能的治疗方法的疗效。(3)组织培养模型： 该核心组件支持用户生成组织培养模型，以研究细胞的发育和 原代脑细胞，包括神经元，星形胶质细胞， 少突胶质细胞、小胶质细胞和内皮细胞。这些细胞来源于基因操纵的啮齿动物，或者它们是 正常细胞暴露于环境刺激，包括药物和毒素。组织培养服务及 干细胞服务密切互动，因为他们共享几种体外方法，并作为一个管道， 推动并帮助设计啮齿动物行为测试。 PMC强调对用户及其工作人员的培训。经过培训的用户可以继续自己的研究 他们可以在实验室中使用这些设备，也可以以较低的成本使用PMC中的设备。这个核心与其他核心相互作用 中心它将与网络中的其他非洲减灾中心交流协议、服务和最佳做法。 与IDDRC使命的相关性：PMC连接了“基因、大脑和行为”的所有三个领域， CHOP/Penn IDDRC（参见总体：中心概述）。核心是为了响应用户的需求而开发的， 一项调查（2014年）强调需要一个以缺碘症为重点的设施，用于研究哺乳动物行为， 一个是解决当前干细胞技术的潜力，包括CRISPR-Cas。 资格：这些服务可供CHOP/UPenn的IDDRC批准用户和 网络中的其他中心。

(PRECLINICAL MODELS CORE (PMC): CORE F)  PROJECT SUMMARY Description: The Preclinical Models Core (PMC) supports IDDRC users who seek new treatments for IDD by studying these disorders in 3 kinds of preclinical models: (1) Rodent models: This Core component characterizes behaviors in mouse models of genetic or acquired disabilities. Measured behaviors include learning, memory, social preference, ultrasonic vocalization, sleep and anxiety. (2) Stem cell models: This Core component assists users to create disease models by generating induced pluripotent stem cells (iPSCs) using standard reprogramming technologies or by genome editing, such as CRISPR-Cas, on established iPSC lines. These cells, some derived from humans with IDD, afford a model in which to scrutinize cellular consequences of genetic disease and to evaluate the efficacy of possible therapies. (3) Tissue culture models: This Core component supports users to generate tissue culture models in which to study the development and behavior of enriched or mixed populations of primary brain cells, including neurons, astrocytes, oligodendroglia, microglia and endothelia. These cells originate in genetically manipulated rodents or they are normal cells exposed to environmental stimuli, including drugs and toxins. The Tissue Culture Service and Stem Cell Service interact closely, since they share several in vitro methodologies and serve as a conduit to drive and help design rodent behavior testing. The PMC emphasizes training of users and their staff. Trained users can continue studies in their own laboratory or they can utilize the equipment in the PMC at reduced cost. This core interacts with others in the Center. It will exchange protocols, services and best practices with other IDDRCs in the Network. Relevance to IDDRC Mission: The PMC bridges all three domains of “Genes, Brain, and Behavior”, the theme of the CHOP/Penn IDDRC (see Overall: Overview of Center). The Core was developed in response to a user survey (2014) that emphasized a need for an IDD-focused facility for the study of mammalian behavior and one to address the potential of current stem cell technologies, including CRISPR-Cas. Eligibility: These services are available both to approved users of the IDDRC at CHOP/UPenn and to users at other Centers in the Network.