Personalized Model System Core

个性化模型系统核心

基本信息

批准号：
10477252
负责人：
Anjaparavanda P Naren
金额：
$ 22.61万
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 2023-06-30
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/10477252
关键词：
Applications Grants Biological Assay Biological Models Biology Cells Center Core Grants Childhood Clinical Cystic Fibrosis Cystic Fibrosis Transmembrane Conductance Regulator Development Disease Duct (organ) structure Embryo Endoderm Fluid Balance Fluids and Secretions Genotype Goals Head Human Interdisciplinary Study International Intestines Lung Medical center Membrane Proteins Methods Microscopic Microscopy Modeling Mutation Myofibroblast Names National Institute of Diabetes and Digestive and Kidney Diseases Nose Organogenesis Organoids Pancreatic duct Patients Pediatric Hospitals Phenotype Physiology Research Research Personnel Resources Services Small Intestines Techniques Testing Therapeutic Therapeutic Studies Tissue Engineering Tissues Training Translational Research clinical investigation cystic fibrosis patients directed differentiation gastrointestinal gastrointestinal transplantation human model human pluripotent stem cell human stem cells in vivo individualized medicine induced pluripotent stem cell induced pluripotent stem cell technology innovation interest intestinal epithelium intestine surgery novel patient population personalized medicine ranpirnase repository sound stem cell derived tissues stem cells 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. 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.

项目概要：该 P30 应用程序的一个关键组件是“个性化模型系统核心”。包括源自肠上皮（肠样）的患者特异性培养物和诱导多能性这些来自肌成纤维细胞的干细胞 (iPSC) 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。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，并将资源分配给全世界的研究人员这是其长期目标。核心是使用源自患者的模型来促进他/她的个性化治疗。将提供 CF 患者特定的肠类药物（小肠和结肠）。 2. 将是。提供特征明确的 CF 患者特异性诱导多能干细胞 (iPSC) 来源的组织具体目标 3. 将为当地、国家和国际检测提供服务和培训。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肠、球体和类器官中的分泌。核心将创造具有重要长期临床意义的资源和健全的服务对针对囊性纤维化患者的特异性治疗的影响该核心将提供各种培训。最先进的创新技术，包括肠道干细胞培养、iPSC 的开发技术，高内涵显微镜等等，对于能够使用这些技术的研究人员来说技术不仅可以研究 CF/CFTR 生物学，还可以研究其他膜蛋白及其相关疾病。