Cardiomyopathy in a dish: Using human inducible pluripotent stem cells to model inherited cardiomyopathies

摘要

Inherited cardiomyopathies including hypertrophic cardiomyopathy (HCM), dilated cardiomyopathies (DCM), arrythmogenic right ventricular cardiomyopathy (ARVC), and other inherited forms of heart failure represent a unique set of genetically defined cardiovascular disease processes. Unraveling the molecular mechanisms of these deadly forms of human heart disease has been challenging, however recent groundbreaking scientific advances in stem cell technology has allowed for the generation of patient-specific human inducible stem cell (hiPSC)-derived cardiomyocytes (hiPSC-derived CMs). hiPSC-derived CMs retain the genetic blueprint of the patient, they can be maintained in culture, and they recapitulate the phenotypic characteristics of the disease in vitro, thus serving as a disease in a dish. This review provides an overview of in vitro modeling of inherited cardiomyopathies using patient-specific hiPSC-derived CMs.