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Personalized Cystic Fibrosis Therapy and Research Center

个性化囊性纤维化治疗和研究中心

基本信息

批准号：
10672706
负责人：
MICHAEL A. HELMRATH
金额：
$ 23.32万
依托单位：
CINCINNATI CHILDRENS HOSP MED CTR
依托单位国家：
美国
项目类别：
财政年份：
2018
资助国家：
美国
起止时间：
2018-08-01 至 2025-06-30
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10672706
关键词：
Address Affect Applications Grants Bedside Testings Biological Assay Biological Models Biology Cell Culture Techniques Cell Line Cells Center Core Grants Childhood Clinical Clinical Trials Colon Cystic Fibrosis Cystic Fibrosis Transmembrane Conductance Regulator Data Dedications Development Disease Dose Duct (organ) structure Embryo Endoderm Fluid Balance Fluids and Secretions Freezing Genotype Goals Human In Vitro Interdisciplinary Study International Intestinal Fibrosis Intestines Lung Medical center Membrane Proteins Methodology Methods Microscopy Modeling Mutation Myofibroblast Names National Institute of Diabetes and Digestive and Kidney Diseases Nose Organ Organogenesis Organoids Pancreas Pancreatic duct Patient-Focused Outcomes Patients Pattern Pediatric Hospitals Phenotype Research Research Personnel Resources Services Small Intestines Techniques Testing Therapeutic Therapeutic Intervention Tissue Engineering Tissues Training Translating Translational Research Underrepresented Minority assay development bench to bedside cell motility clinical investigation clinically relevant cystic fibrosis patients directed differentiation drug discovery gastrointestinal gastrointestinal transplantation human pluripotent stem cell human stem cells implantation improved in vivo in vivo Model individualized medicine induced pluripotent stem cell induced pluripotent stem cell technology innovation interest intestinal epithelium intestine surgery multidisciplinary mutant next generation novel patient population personalized medicine repository sound stem cell derived tissues stem cells three dimensional structure tool

项目摘要

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. To provide well- characterized CF patient-specific enteroids (small bowel and colonic) to local, national, and international investigators interested in personalized model systems or CF/CFTR-related research. Specific Aim 2. To provide well-characterized CF patient-specific iPSC-derived tissue from myofibroblasts to local, national, and international investigators interested in personalized model systems. Specific Aim 3. To provide assay services and training to local, national, and international investigators interested in personalized model systems to study the functional involvement of CFTR fluid secretion in intestinal enteroids and spheroids. 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.

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”。这些模型将包括源自肠上皮（肠样）的患者特异性培养物和诱导多能性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 功能和体液稳态，获得的信息将用于个性化治疗方案。这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 CF/CFTR 研究，并将资源分发给世界各地的研究人员。该核心的长期目标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. 提供良好的向当地、国家和国际描述 CF 患者特异性肠样物质（小肠和结肠）对个性化模型系统或 CF/CFTR 相关研究感兴趣的研究人员。具体目标 2. 至向地方、国家和地区提供来自肌成纤维细胞的特征明确的 CF 患者特异性 iPSC 衍生组织对个性化模型系统感兴趣的国际研究人员。具体目标 3. 提供检测为对个性化模型感兴趣的本地、国家和国际研究人员提供服务和培训系统研究肠肠类和球体中 CFTR 液体分泌的功能参与。核心将创造具有重要长期临床影响的资源和完善的服务针对囊性纤维化的患者特异性疗法。该核心将提供各种最先进和创新技术，包括肠道干细胞培养物的开发、iPSC 技术、高仅举几例，研究人员将能够使用这些技术来研究内容显微镜 CF/CFTR 生物学，但其他膜蛋白及其相关疾病。