Human-induced pluripotent stem cell approaches to model inborn and acquired metabolic heart diseases

被引:10
|
作者
Chanana, Anita M. [1 ,2 ]
Rhee, June-Wha [1 ,2 ]
Wu, Joseph C. [1 ,2 ,3 ,4 ]
机构
[1] Stanford Univ, Sch Med, Stanford Cardiovasc Inst, Stanford, CA 94305 USA
[2] Stanford Univ, Sch Med, Dept Med, Div Cardiol, Stanford, CA 94305 USA
[3] Stanford Univ, Sch Med, Dept Radiol, Stanford, CA 94305 USA
[4] Stanford Univ, Sch Med, Inst Stem Cell Biol & Regenerat Med, Stanford, CA 94305 USA
基金
美国国家卫生研究院;
关键词
cardiomyocytes; induced pluripotent stem cells; metabolic heart disease; POMPE DISEASE; BARTH-SYNDROME; CARDIOMYOPATHY; PATIENT; FIBROBLASTS; GENERATION; PHENOTYPE;
D O I
10.1097/HCO.0000000000000277
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Purpose of review The article provides an overview of advances in the induced pluripotent stem cell field to model cardiomyopathies of inherited inborn errors of metabolism and acquired metabolic syndromes in vitro. Recent findings Several inborn errors of metabolism have been studied using disease in a dish' models, including Pompe disease, Danon disease, Fabry disease, and Barth syndrome. Disease phenotypes of complex metabolic syndromes, such as diabetes mellitus and aldehyde dehydrogenase 2 deficiency, have also been observed. Summary Differentiation of patient and disease-specific induced pluripotent stem cell-derived cardiomyocytes has provided the capacity to model deleterious cardiometabolic diseases to understand molecular mechanisms, perform drug screens, and identify novel drug targets.
引用
收藏
页码:266 / 274
页数:9
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