Personalized Model System Core

项目概要： 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 包括来源于肠上皮细胞（类肠上皮细胞）的患者特异性培养物和诱导的多能 来自肌成纤维细胞的干细胞（iPSC）。这些患者特异性培养物将用于研究CFTR功能 和体液平衡，所获得的信息将用于个性化治疗方案。 个性化模型系统核心将开发和储存这些模型，开发和验证测定 用于CF/CFTR研究，并将资源分发给世界各地的研究人员。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CF患者特定的肠液（小肠和结肠）。具体目标2。将是 提供充分表征的CF患者特异性诱导多能干细胞（iPSC）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 对个性化模型系统感兴趣的研究者，以研究CFTR液体的功能参与 肠类肠、球状体和类器官中的分泌。 核心将创造资源和良好的服务，具有重要的长期临床意义， 对囊性纤维化患者特异性治疗的影响。核心将提供各种培训， 国家的最先进的和创新的技术，包括肠道干细胞培养，iPSC的发展 技术，高内容显微镜，仅举几例，研究人员谁将能够使用这些 这些技术不仅可以研究CF/CFTR生物学，还可以研究其他膜蛋白及其相关疾病。

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.