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Genetic and Stem Cell Model of Cardiac Metabolic Disease

心脏代谢疾病的遗传和干细胞模型

基本信息

批准号：
9893900
负责人：
THOMAS QUERTERMOUS
金额：
$ 75.09万
依托单位：
STANFORD UNIVERSITY
依托单位国家：
美国
项目类别：
财政年份：
2019
资助国家：
美国
起止时间：
2019-04-01 至 2023-03-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/9893900
关键词：
Affect Autophagocytosis Basic Science Bioinformatics Biometry CRISPR/Cas technology Cardiac Cardiac Myocytes Cardiomyopathies Cardiovascular Diseases Cause of Death Cell Line Cholesterol Clinic Clinical Clinical Research Complex Coronary Arteriosclerosis DNA Data Defect Development Diabetes Mellitus Disease Disease susceptibility Endocrinology Endothelial Cells Endothelium Enrollment Evolution Exhibits Exposure to Functional disorder Genes Genetic Genomics Genotype Goals Grant Human Hyperglycemia Hypertension Impairment Individual Interdisciplinary Study Left Left Ventricular Ejection Fraction Metabolic Metabolic Diseases Mitochondria Modeling Molecular Molecular Genetics Molecular Profiling Myocardial dysfunction Names Non-Insulin-Dependent Diabetes Mellitus Patients Physical activity Population Positioning Attribute Predisposition Relaxation Research Personnel Risk Risk Factors Sampling Selection Criteria Single Nucleotide Polymorphism Smoking Structure Susceptibility Gene Training Programs Translational Research Type 2 diabetic Ventricular base cardiometabolism cardiovascular disorder risk design diabetic diabetic cardiomyopathy diabetic patient disease phenotype effective therapy ethnic diversity gender diversity genetic information genome editing genome sequencing genomic data high risk induced pluripotent stem cell insight molecular phenotype novel patient subsets personalized approach precision genetics pressure randomized trial recruit response stem cell biology stem cell model targeted treatment transcriptome sequencing treatment strategy whole genome

项目摘要

PROJECT SUMMARY/ABSTRACT Type 2 diabetes (T2D) is a long-term metabolic disorder affecting 12% of the US population. It is a leading cause of death nationwide, primarily due to associated cardiovascular disease (CVD, >65% of patients). While CVD risk factors include high cholesterol, hypertension, and smoking, a subset of patients suffer from myocardial dysfunction, a term named type 2 diabetic cardiomyopathy (T2DCM), which suggest factors within the cardiac myocyte itself may give rise to detrimental cardiac remodeling associated with diabetes. Despite the obvious importance of T2DCM, there is currently no specific effective treatment for it and a deep understanding of this complex disease at the molecular level is lacking. Hence, resolving the contributing mechanisms of T2DCM is a pressing goal of basic and translational research. The recent advent of new technological breakthroughs, such as patient-specific human induced pluripotent stem cells (iPSCs) and genome editing, provides an unprecedented opportunity to study associations between genetic variability and disease susceptibility. The overarching goal of our multi-PI R01 grant is to understand the underlying mechanisms of T2DCM using patient-specific iPSC-derived cardiomyocytes and endothelial cells from T2D patients and to understand individual susceptibility to disease development. We have assembled a team of highly accomplished clinicians and researchers in cardiac stem cell biology, genomics, molecular genetics, biostatistics and bioinformatics. We are well positioned to achieve the project goals within five years. Our proposal will enable a novel personalized approach to better understand the mechanisms underlying T2DCM that could ultimately revolutionize treatment strategies.

项目总结/文摘