title: Human-Induced Pluripotent Stem Cell-Based Modeling of Cardiac Storage Disorders.
abstract: The aim of this study is to review the published human-induced pluripotent stem cell-derived cardiomyocyte (hiPSC-CM) models of cardiac storage disorders and to evaluate the limitations and future applications of this technology.
  
  
  
  Several cardiac storage disorders (CSDs) have been modeled using patient-specific hiPSC-CMs, including Anderson-Fabry disease, Danon disease, and Pompe disease. These models have shown that patient-specific hiPSC-CMs faithfully recapitulate key phenotypic features of CSDs and respond predictably to pharmacologic manipulation. hiPSC-CMs generated from patients with CSDs are representative models of the patient disease state and can be used as an in vitro system for the study of human cardiomyocytes. While these models suffer from several limitations, they are likely to play an important role in future mechanistic studies of cardiac storage disorders and the development of targeted therapeutics for these diseases.
authors.affiliation: Department of Medicine, Division of Cardiology, University of California San Diego, 9500 Gilman Drive, Biomedical Research Facility, Room 1217 AA, La Jolla, CA, 92093, USA.
authors.forename: Bradley C
authors.initials: BC
authors.lastname: Nelson
authors.affiliation: Department of Medicine, Division of Cardiology, University of California San Diego, 9500 Gilman Drive, Biomedical Research Facility, Room 1217 AA, La Jolla, CA, 92093, USA.
authors.forename: Sherin I
authors.initials: SI
authors.lastname: Hashem
authors.affiliation: Department of Medicine, Division of Cardiology, University of California San Diego, 9500 Gilman Drive, Biomedical Research Facility, Room 1217 AA, La Jolla, CA, 92093, USA. eradler@ucsd.edu.
authors.forename: Eric D
authors.initials: ED
authors.lastname: Adler
mesh_terms: D000795:Fabry Disease; D006009:Glycogen Storage Disease Type II / Q000473:pathology; D052120:Glycogen Storage Disease Type IIb / Q000473:pathology; D006331:Heart Diseases / Q000473:pathology*; D006801:Humans; D057026:Induced Pluripotent Stem Cells / Q000473:pathology*; D008661:Metabolism, Inborn Errors / Q000473:pathology*; D009083:Mucopolysaccharidoses / Q000473:pathology; D032383:Myocytes, Cardiac / Q000473:pathology*; D013106:Sphingolipidoses / Q000473:pathology
keywords: Cardiac storage disorders; Cardiomyocyte; Disease modeling; Induced pluripotent stem cells