Personalized Model System Core

个性化模型系统核心

基本信息

批准号：
10249242
负责人：
Anjaparavanda P Naren
金额：
$ 22.63万
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/10249242
关键词：
Applications Grants Biological Assay Biological Models Biology Cell Culture Techniques 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 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

Applications Grants Biological Assay Biological Models Biology Cell Culture Techniques 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 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

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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.

项目摘要：该P30应用程序的关键组成部分是“个性化模型系统核心”。这些模型会包括源自肠上皮（Enteroids）的患者特异性培养物和诱导的多能来自肌纤维细胞的干细胞（IPSC）。这些患者特定的培养物将用于研究CFTR功能和流体稳态以及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库存这些模型，开发和验证测定法用于CF/CFTR研究，并将资源分配给全球研究人员。这个长期目标核心是使用从患者衍生的模型来促进他/她的个性化疗法。具体目标1。将提供CF患者特异性的肠to（小肠和结肠）。具体目标2。提供良好特征的CF患者特异性诱导多能干细胞（IPSC）衍生的组织肌纤维细胞。特定目标3。将向本地，国家和国际提供评估服务和培训对个性化模型系统感兴趣的研究人员研究CFTR流体的功能参与肠内肠，球体和器官的分泌。核心将创建具有重要长期临床的资源和声音服务对囊性纤维化的患者特异性疗法的影响。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的开发，IPSCS 对于能够使用这些的研究人员来说，技术，高素质显微镜，仅举几例不仅研究CF/CFTR生物学，还研究其他膜蛋白及其相关疾病的技术。