Disease Modeling Core

疾病建模核心

基本信息

批准号：
10646158
负责人：
Holger A. Russ
金额：
$ 17.39万
依托单位：
UNIVERSITY OF COLORADO DENVER
依托单位国家：
美国
项目类别：
财政年份：
2020
资助国家：
美国
起止时间：
2020-04-01 至 2025-03-31
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10646158
关键词：
Adult Beta Cell Biological Assay Biological Models CRISPR/Cas technology Cell Differentiation process Cell Line Cell model Cells Clinical Clustered Regularly Interspaced Short Palindromic Repeats Communities Complex Diabetes Mellitus Disease Disease model Ensure Enzyme-Linked Immunosorbent Assay Fingerprint Gene Expression Generations Genes Genetic Models Genome engineering Goals Human Human Cell Line Immunotherapy In Vitro Individual Infrastructure Institution Karyotype determination procedure Laboratories Libraries Liver Luciferases Medical Microsatellite Repeats Minisatellite Repeats Mission Modeling Molecular Mycoplasma Organoids Pancreas Pancreatic duct Patients Peripheral Blood Mononuclear Cell Pluripotent Stem Cells Point Mutation Procedures Protocols documentation Quality Control Quantitative Reverse Transcriptase PCR Reagent Reporter Reporter Genes Reproducibility Research Research Personnel Resources Sampling Scientist Secure Services Standardization System Technology Testing Time Tissue Model Tissue Procurements Tissues Training Transplantation United States National Institutes of Health Validation adult stem cell assay development biobank cell repository cell type cost cost effective data sharing design directed differentiation functional genomics gene correction genome editing human adult stem cell human disease human embryonic stem cell human model human stem cells human tissue humanized mouse in vitro Model in vivo induced pluripotent stem cell innovation member mouse model novel overexpression pluripotency pre-clinical programs searchable database small hairpin RNA stem cell model stem cell technology stem cells translational potential translational scientist type I and type II diabetes

项目摘要

DISEASE MODELING CORE PROJECT SUMMARY/ABSTRACT The overall mission of the Disease Modeling Core (DM core) is to provide diabetes researchers with access to novel human stem cell-derived in vitro cell models for investigating cellular and molecular features of both Type 1 and Type 2 diabetes. Recent progress in stem cell, organoid culture, gene editing and directed differentiation technologies has afforded opportunities to develop state-of-the-art pre-clinical human models. However, because of the complex experimental protocols and specialized reagents used, together with the considerable up-front costs and time required to develop these sophisticated stem cell-based models, it is not practical nor cost-effective to develop these models in individual laboratories. The goal of DM core is to provide the expertise, infrastructure and access to novel human model systems and technologies to DRC investigators. To help overcome the obstacles faced by basic scientists and clinical researchers who wish to establish human stem cell approaches in their laboratories, we will provide expertise, resources and training in start-of-the-art stem cell technologies together with rigorous quality control testing, validation standardization and authentication all model platforms and reagents. This goal will be achieved in three ways: 1) providing access and training in current and emerging pluripotent and adult stem cell technologies for in vitro human disease modeling, including the use of organoids; 2) providing access, training and implementation of genome editing technology, directed cell differentiation/programming, and gene expression systems; and 3) providing validation, authentication and data sharing of all DM-related technologies. Thus, the overall mission of this core is to facilitate access, generation and usage of novel human stem cell-derived in vitro models to promote basic and translational innovative diabetes research at UC AMC.

疾病建模核心项目概要/摘要疾病建模核心（DM 核心）的总体使命是为糖尿病研究人员提供新型人类干细胞衍生的体外细胞模型，用于研究两种类型的细胞和分子特征 1型和2型糖尿病。干细胞、类器官培养、基因编辑和定向分化的最新进展技术为开发最先进的临床前人体模型提供了机会。然而，由于复杂的实验方案和使用的专门试剂，以及大量的开发这些复杂的基于干细胞的模型所需的前期成本和时间，既不实用也不可行在各个实验室开发这些模型具有成本效益。 DM core 的目标是提供专业知识、基础设施以及为刚果民主共和国调查人员提供新型人体模型系统和技术的机会。帮助克服希望建立人类干细胞的基础科学家和临床研究人员所面临的障碍他们实验室的方法，我们将提供最先进的干细胞方面的专业知识、资源和培训技术以及严格的质量控制测试、验证标准化和认证所有模型平台和试剂。这一目标将通过三种方式实现：1）提供当前和未来的访问和培训用于体外人类疾病模型的新兴多能和成体干细胞技术，包括使用类器官； 2）提供基因组编辑技术、定向细胞的获取、培训和实施分化/编程和基因表达系统； 3) 提供验证、认证和数据共享所有 DM 相关技术。因此，该核心的总体使命是促进访问、生成和使用新型人类干细胞体外模型来促进基础和转化创新 UC AMC 的糖尿病研究。