Correction of human phospholamban R14del mutation associated with cardiomyopathy using targeted nucleases and combination therapy

Targeted Gene Correction Minimally Impacts Whole-Genome Mutational Load in Human-Disease-Specific Induced Pluripotent Stem Cell Clones

(2014) Keiichiro Suzuki et al.   Cell Stem Cell

Low Incidence of Off-Target Mutations in Individual CRISPR-Cas9 and TALEN Targeted Human Stem Cell Clones Detected by Whole-Genome Sequencing

(2014) Adrian Veres et al.   Cell Stem Cell

Whole-Genome Sequencing Analysis Reveals High Specificity of CRISPR/Cas9 and TALEN-Based Genome Editing in Human iPSCs

(2014) Cory Smith et al.   Cell Stem Cell

Genome Editing of Isogenic Human Induced Pluripotent Stem Cells Recapitulates Long QT Phenotype for Drug Testing

(2014) Yongming Wang et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Modeling CVD in Human Pluripotent Cells by Genome Editing∗

(2014) Roger J. Hajjar et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Modeling the mitochondrial cardiomyopathy of Barth syndrome with induced pluripotent stem cell and heart-on-chip technologies

(2014) Gang Wang et al.   NATURE MEDICINE

Mybpc3 gene therapy for neonatal cardiomyopathy enables long-term disease prevention in mice

(2014) Giulia Mearini et al.   Nature Communications

Abnormal Calcium Handling Properties Underlie Familial Hypertrophic Cardiomyopathy Pathology in Patient-Specific Induced Pluripotent Stem Cells

(2013) Feng Lan et al.   Cell Stem Cell

Genetic analysis in 418 index patients with idiopathic dilated cardiomyopathy: overview of 10 years' experience

(2013) Karin Y. van Spaendonck-Zwarts et al.   EUROPEAN JOURNAL OF HEART FAILURE

Studying arrhythmogenic right ventricular dysplasia with patient-specific iPSCs

(2013) Changsung Kim et al.   NATURE

Dilated cardiomyopathy: the complexity of a diverse genetic architecture

(2013) Ray E. Hershberger et al.   Nature Reviews Cardiology

Modeling of Friedreich ataxia-related iron overloading cardiomyopathy using patient-specific-induced pluripotent stem cells

(2013) Yee-Ki Lee et al.   PFLUGERS ARCHIV-EUROPEAN JOURNAL OF PHYSIOLOGY

Allele-Specific Silencing of Mutant Myh6 Transcripts in Mice Suppresses Hypertrophic Cardiomyopathy

(2013) Jianming Jiang et al.   SCIENCE

Recurrent and founder mutations in the Netherlands—Phospholamban p.Arg14del mutation causes arrhythmogenic cardiomyopathy

(2013) P. A. van der Zwaag et al.   Netherlands Heart Journal

Small Molecule-Mediated Directed Differentiation of Human Embryonic Stem Cells Toward Ventricular Cardiomyocytes

(2013) Ioannis Karakikes et al.   Stem Cells Translational Medicine

Disease characterization using LQTS-specific induced pluripotent stem cells

(2012) Toru Egashira et al.   CARDIOVASCULAR RESEARCH

Cardiomyocytes Derived From Pluripotent Stem Cells Recapitulate Electrophysiological Characteristics of an Overlap Syndrome of Cardiac Sodium Channel Disease

(2012) Richard P. Davis et al.   CIRCULATION

Phospholamban R14del mutation in patients diagnosed with dilated cardiomyopathy or arrhythmogenic right ventricular cardiomyopathy: evidence supporting the concept of arrhythmogenic cardiomyopathy

(2012) Paul A. van der Zwaag et al.   EUROPEAN JOURNAL OF HEART FAILURE

Reference Maps of Human ES and iPS Cell Variation Enable High-Throughput Characterization of Pluripotent Cell Lines

(2011) Christoph Bock et al.   CELL

In vitro Modeling of Ryanodine Receptor 2 Dysfunction Using Human Induced Pluripotent Stem Cells

(2011) Azra Fatima et al.   CELLULAR PHYSIOLOGY AND BIOCHEMISTRY

Model for long QT syndrome type 2 using human iPS cells demonstrates arrhythmogenic characteristics in cell culture

(2011) A. L. Lahti et al.   Disease Models & Mechanisms

Dantrolene rescues arrhythmogenic RYR2 defect in a patient-specific stem cell model of catecholaminergic polymorphic ventricular tachycardia

(2011) Christian B. Jung et al.   EMBO Molecular Medicine

Drug evaluation in cardiomyocytes derived from human induced pluripotent stem cells carrying a long QT syndrome type 2 mutation

(2011) Elena Matsa et al.   EUROPEAN HEART JOURNAL

Cardiomyocytes generated from CPVTD307H patients are arrhythmogenic in response to β-adrenergic stimulation

(2011) Atara Novak et al.   JOURNAL OF CELLULAR AND MOLECULAR MEDICINE

The human phospholamban Arg14-deletion mutant localizes to plasma membrane and interacts with the Na/K-ATPase

(2011) Kobra Haghighi et al.   JOURNAL OF MOLECULAR AND CELLULAR CARDIOLOGY

Hotspots of aberrant epigenomic reprogramming in human induced pluripotent stem cells

(2011) Ryan Lister et al.   NATURE

Using induced pluripotent stem cells to investigate cardiac phenotypes in Timothy syndrome

(2011) Masayuki Yazawa et al.   NATURE

Modelling the long QT syndrome with induced pluripotent stem cells

(2011) Ilanit Itzhaki et al.   NATURE

Genetic engineering of human pluripotent cells using TALE nucleases

(2011) Dirk Hockemeyer et al.   NATURE BIOTECHNOLOGY

Highly Efficient Reprogramming to Pluripotency and Directed Differentiation of Human Cells with Synthetic Modified mRNA

(2010) Luigi Warren et al.   Cell Stem Cell

The genetics of dilated cardiomyopathy

(2010) Lisa Dellefave et al.   CURRENT OPINION IN CARDIOLOGY

A TALE nuclease architecture for efficient genome editing

(2010) Jeffrey C Miller et al.   NATURE BIOTECHNOLOGY

Patient-Specific Induced Pluripotent Stem-Cell Models for Long-QT Syndrome

(2010) Alessandra Moretti et al.   NEW ENGLAND JOURNAL OF MEDICINE

Genetic deletion of arginine 14 in phospholamban causes dilated cardiomyopathy with attenuated electrocardiographic R amplitudes

(2009) Maximilian G. Posch et al.   HEART RHYTHM

Molecular characterization and functional properties of cardiomyocytes derived from human inducible pluripotent stem cells

(2009) Igal Germanguz et al.   JOURNAL OF CELLULAR AND MOLECULAR MEDICINE

A human phospholamban promoter polymorphism in dilated cardiomyopathy alters transcriptional regulation by glucocorticoids

(2008) Kobra Haghighi et al.   HUMAN MUTATION