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Disease models

Induced pluripotent stem (iPS) cell-derived models of familial hypertrophic cardiomyopathy (HCM)

Patient-derived iPS cell models of familial HCM could help identify new treatments for the disease. HCM is a hereditary heart disease caused by many distinct mutations that affect cardiac muscle function. iPS cell-derived cardiomyocytes were generated from fibroblasts from patients with HCM carrying a mutation in myosin heavy chain 7 cardiac muscle-b (MYH7). These cardiomyocytes showed disease-associated phenotypes including increased cell size and multinucleation compared with cardiomyocytes derived from healthy subjects and showed abnormal calcium signaling. In these cells, a calcium channel blocker decreased hypertrophy compared with no treatment. Ongoing work includes developing a patient-specific iPS cell disease library and screening for HCM therapeutics.

SciBX 6(4); doi:10.1038/scibx.2013.97
Published online Jan. 31, 2013

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Lan, F. et al. Cell Stem Cell; published online Jan. 3, 2013;
Contact: Joseph C. Wu, Stanford University School of Medicine, Stanford, Calif.