title: Genomic correction of familial cardiomyopathy in human engineered cardiac tissues.
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.
authors.affiliation: Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, One Gustave L. Levy Place, PO Box 1030, New York, NY 10029, USA.
authors.forename: Francesca
authors.initials: F
authors.lastname: Stillitano
authors.affiliation: Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, One Gustave L. Levy Place, PO Box 1030, New York, NY 10029, USA.
authors.forename: Irene C
authors.initials: IC
authors.lastname: Turnbull
authors.affiliation: Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, One Gustave L. Levy Place, PO Box 1030, New York, NY 10029, USA.|Department of Medicine, Division of Cardiovascular Medicine, Stanford Cardiovascular Institute, Stanford, CA 94305, USA.
authors.forename: Ioannis
authors.initials: I
authors.lastname: Karakikes
authors.affiliation: Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, One Gustave L. Levy Place, PO Box 1030, New York, NY 10029, USA.
authors.forename: Mathieu
authors.initials: M
authors.lastname: Nonnenmacher
authors.affiliation: Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, One Gustave L. Levy Place, PO Box 1030, New York, NY 10029, USA.
authors.forename: Peter
authors.initials: P
authors.lastname: Backeris
authors.affiliation: Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, One Gustave L. Levy Place, PO Box 1030, New York, NY 10029, USA.|Sorbonne Universités, UPMC Univ Paris 06, AP-HP, Institute of Cardiometabolism and Nutrition (ICAN), Pitié-Salpêtrière Hospital, Paris F-75013, France.
authors.forename: Jean-Sébastien
authors.initials: JS
authors.lastname: Hulot
authors.affiliation: Department of Pharmacology, University of Cincinnati, Cincinnati, OH 45267-0575, USA.
authors.forename: Evangelia G
authors.initials: EG
authors.lastname: Kranias
authors.affiliation: 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.
authors.forename: Roger J
authors.initials: RJ
authors.lastname: Hajjar
authors.affiliation: Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, One Gustave L. Levy Place, PO Box 1030, New York, NY 10029, USA.
authors.forename: Kevin D
authors.initials: KD
authors.lastname: Costa
mesh_terms: D002135:Calcium-Binding Proteins; D009202:Cardiomyopathies; D023281:Genomics; D006801:Humans; D009154:Mutation
chemical_list: D002135:Calcium-Binding Proteins
keywords: Dilated cardiomyopathy; Engineered heart tissue; Gene editing; Induced pluripotent stem cells; Phospholamban