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Eur Heart J. 2016 Nov 14;37(43):3282-3284. Epub 2016 Jul 22.

Genomic correction of familial cardiomyopathy in human engineered cardiac tissues.

Author information

1
Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, One Gustave L. Levy Place, PO Box 1030, New York, NY 10029, USA.
2
Department of Medicine, Division of Cardiovascular Medicine, Stanford Cardiovascular Institute, Stanford, CA 94305, USA.
3
Sorbonne Universités, UPMC Univ Paris 06, AP-HP, Institute of Cardiometabolism and Nutrition (ICAN), Pitié-Salpêtrière Hospital, Paris F-75013, France.
4
Department of Pharmacology, University of Cincinnati, Cincinnati, OH 45267-0575, USA.
5
Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, One Gustave L. Levy Place, PO Box 1030, New York, NY 10029, USA roger.hajjar@mssm.edu.

Abstract

In this study, we used three-dimensional human engineered cardiac tissue technology to directly show that phospholamban (PLN) R14del mutation impairs cardiac contractility and to demonstrate restoration of contractile properties with targeted genetic correction of this inheritable form of dilated cardiomyopathy.

KEYWORDS:

Dilated cardiomyopathy; Engineered heart tissue; Gene editing; Induced pluripotent stem cells; Phospholamban

PMID:
27450564
DOI:
10.1093/eurheartj/ehw307
[Indexed for MEDLINE]

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