Personalized Model System Core

项目总结： 这个P30应用程序的一个关键组件是“个性化模型系统核心”。模特们将会 包括来自肠上皮(肠样)的患者特异性培养和诱导的多能性 来自肌成纤维细胞的干细胞(IPSCs)。这些患者特有的培养将用于研究CFTR的功能 和液体动态平衡，所获得的信息将用于个性化治疗选择。 个性化模型系统核心将开发和存储这些模型，开发和验证分析 用于CF/CFTR研究，并将资源分发给世界各地的研究人员。这样做的长期目标是 核心是使用来自患者的模型来促进他/她的个性化治疗。具体目标1. 将提供针对CF患者的肠样体(小肠和结肠)。具体目标2.将是 提供特征性良好的CF患者特异性诱导多能干细胞(IPSCs)来源的组织 肌成纤维细胞。具体目标3.将为当地、国家和国际提供化验服务和培训 研究人员对个性化模型系统感兴趣，以研究CFTR液体的功能参与 肠类、球体和器官类的分泌物。 核心将创造具有重要长期临床意义的资源和完善的服务 对囊性纤维化患者特异性治疗的启示。该核心将提供各种不同的培训 最先进和创新的技术，包括开发肠道干细胞培养，IPSCs 技术，高含量显微镜，举几个例子，给将能够使用这些技术的研究人员 不仅研究CF/CFTR生物学，而且研究其他膜蛋白及其相关疾病的技术。

PROJECT SUMMARY: A key component of this P30 application is the ‘Personalized Model System Core’. The models will include the patient-specific cultures derived from intestinal epithelia (enteroids) and the induced pluripotent stem cells (iPSCs) from myofibroblasts. These patient specific cultures will be used to study CFTR function and fluid homeostasis, and the information obtained will be used towards personalized treatment options. The Personalized Model System core will develop and bank these models, develop and validate the assays for CF/CFTR research, and distribute the resources to researchers worldwide. The long-term goal of this core is to use models derived from the patient to facilitate his/her personalized therapy. Specific Aim 1. Will be to provide CF patient-specific enteroids (small bowel and colonic). Specific Aim 2. Will be to provide well-characterized CF patient-specific induced pluripotent stem cells (iPSCs)-derived tissue from myofibroblasts. Specific Aim 3. Will provide assay services and training to local, national and international investigators interested in personalized model system to study the functional involvement of CFTR fluid secretion in intestinal enteroids, spheroids and organoids. The Core will create resources and sound services that have important long term clinical implications toward patient-specific therapies for cystic fibrosis. The core will provide training in a variety of state-of-art and innovative techniques including the development of intestinal stem cells cultures, iPSCs technology, high content microscopy, to name a few, to researchers who will be able to use these techniques to study not only CF/CFTR biology but other membrane proteins and their associated diseases.