Modeling inherited cardiac disorders: A cell is worth a thousand genes

Karim Sallam, Kazuki Kodo, Joseph C. Wu

Research output: Contribution to journalReview article

24 Citations (Scopus)

Abstract

Advances in the understanding and treatment of cardiac disorders have been thwarted by the inability to study beating human cardiac cells in vitro. Induced pluripotent stem cells (iPSCs) bypass this hurdle by enabling the creation of patient-specific iPSC-derived cardiomyocytes (iPSC-CMs). These cells provide a unique platform to study cardiac diseases in vitro, especially hereditary cardiac conditions. To date, iPSC-CMs have been used to successfully model arrhythmic disorders, showing excellent recapitulation of cardiac channel function and electrophysiologic features of long QT syndrome types 1, 2, 3, and 8, and catecholaminergic polymorphic ventricular tachycardia (CPVT). Similarly, iPSC-CM models of dilated cardiomyopathy (DCM) and hypertrophic cardiomyopathy (HCM) have shown robust correlation of predicted morphologic, contractile, and electrical phenotypes. In addition, iPSC-CMs have shown some features of the respective phenotypes for arrhythmogenic right ventricular dysplasia/cardiomy-opathy (ARVD/C), LEOPARD syndrome, Pompe's disease, and Friedriech's ataxia. In this review, we examine the progress of utilizing iPSC-CMs as a model for cardiac conditions and analyze the potential for the platform in furthering the biology and treatment of cardiac disorders.

Original languageEnglish
Pages (from-to)784-794
Number of pages11
JournalCirculation Journal
Volume78
Issue number4
DOIs
Publication statusPublished - 2014
Externally publishedYes

Fingerprint

Induced Pluripotent Stem Cells
Genes
Romano-Ward Syndrome
LEOPARD Syndrome
Glycogen Storage Disease Type II
Arrhythmogenic Right Ventricular Dysplasia
Phenotype
Hypertrophic Cardiomyopathy
Dilated Cardiomyopathy
Ataxia
Cardiac Myocytes
Heart Diseases
Therapeutics

Keywords

  • Channelopathy
  • Dilated cardiomyopathy
  • Drug discovery
  • Human-induced pluripotent stem cells
  • Hypertrophic cardiomyopathy

ASJC Scopus subject areas

  • Cardiology and Cardiovascular Medicine

Cite this

Modeling inherited cardiac disorders : A cell is worth a thousand genes. / Sallam, Karim; Kodo, Kazuki; Wu, Joseph C.

In: Circulation Journal, Vol. 78, No. 4, 2014, p. 784-794.

Research output: Contribution to journalReview article

Sallam, Karim ; Kodo, Kazuki ; Wu, Joseph C. / Modeling inherited cardiac disorders : A cell is worth a thousand genes. In: Circulation Journal. 2014 ; Vol. 78, No. 4. pp. 784-794.
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