Cardiomyopathy in a Dish: Using Human Inducible Pluripotent Stem Cells to Model Inherited Cardiomyopathies

• Human inducible pluripotent stem cells (hiPSCs) are from a somatic source and retain the patient genotype.
• hiPSC-derived cardiomyocytes (CMs) serve as a disease model in a dish.
• Inherited cardiomyopathies including dilated cardiomyopathy, hypertrophic cardiomyopathy, and arrythmogenic right ventricular dysplasia have been modeled.
• hiPSC-derived CM models of inherited cardiomyopathies have allowed for understanding disease mechanism and testing therapies.

Inherited cardiomyopathies, including hypertrophic cardiomyopathy, dilated cardiomyopathies, arrythmogenic right ventricular cardiomyopathy, 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, but recent groundbreaking scientific advances in stem cell technology have allowed for the generation of patient-specific human inducible stem cell (hiPSC)–derived cardiomyocytes (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 with the use of patient-specific hiPSC-derived CMs.

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