Allele-specific ablation rescues electrophysiological abnormalities in a human iPS cell model of long-QT syndrome with a CALM2 mutation

A Precision Medicine Approach to the Rescue of Function on Malignant Calmodulinopathic Long-QT SyndromeNovelty and Significance

(2016) Worawan B. Limpitikul et al.   CIRCULATION RESEARCH

Arrhythmogenic Calmodulin Mutations Affect the Activation and Termination of Cardiac Ryanodine Receptor-mediated Ca2+Release

(2015) Mads T. Søndergaard et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

In vivo gene editing in dystrophic mouse muscle and muscle stem cells

(2015) M. Tabebordbar et al.   SCIENCE

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

(2015) C. E. Nelson et al.   SCIENCE

Functional Gene Correction for Cystic Fibrosis in Lung Epithelial Cells Generated from Patient iPSCs

(2015) Amy L. Firth et al.   Cell Reports

Efficient CRISPR-Cas9-Mediated Generation of Knockin Human Pluripotent Stem Cells Lacking Undesired Mutations at the Targeted Locus

(2015) Florian T. Merkle et al.   Cell Reports

Precise Correction of the Dystrophin Gene in Duchenne Muscular Dystrophy Patient Induced Pluripotent Stem Cells by TALEN and CRISPR-Cas9

(2015) Hongmei Lisa Li et al.   Stem Cell Reports

Genetic and Clinical Advances in Congenital Long QT Syndrome

(2014) Yuka Mizusawa et al.   CIRCULATION JOURNAL

Divergent Regulation of Ryanodine Receptor 2 Calcium Release Channels by Arrhythmogenic Human Calmodulin Missense Mutants

(2014) Hyun Seok Hwang et al.   CIRCULATION RESEARCH

Novel Calmodulin Mutations Associated With Congenital Arrhythmia Susceptibility

(2014) N. Makita et al.   Circulation-Cardiovascular Genetics

Calmodulin mutations associated with long QT syndrome prevent inactivation of cardiac L-type Ca2+ currents and promote proarrhythmic behavior in ventricular myocytes

(2014) Worawan B. Limpitikul et al.   JOURNAL OF MOLECULAR AND CELLULAR CARDIOLOGY

Chemically defined generation of human cardiomyocytes

(2014) Paul W Burridge et al.   NATURE METHODS

Efficient genome modification by CRISPR-Cas9 nickase with minimal off-target effects

(2014) Bin Shen et al.   NATURE METHODS

Seamless modification of wild-type induced pluripotent stem cells to the natural CCR5Δ32 mutation confers resistance to HIV infection

(2014) Lin Ye et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Arrhythmogenic Calmodulin Mutations Disrupt Intracellular Cardiomyocyte Ca 2+ Regulation by Distinct Mechanisms

(2014) Guo Yin et al.   Journal of the American Heart Association

Calmodulin Mutations Associated With Recurrent Cardiac Arrest in Infants

(2013) Lia Crotti et al.   CIRCULATION

Calmodulin in a Heartbeat

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Generation of an ICF Syndrome Model by Efficient Genome Editing of Human Induced Pluripotent Stem Cells Using the CRISPR System

(2013) Takuro Horii et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Identification and characterization of a transient outward K+ current in human induced pluripotent stem cell-derived cardiomyocytes

(2013) Jonathan M. Cordeiro et al.   JOURNAL OF MOLECULAR AND CELLULAR CARDIOLOGY

A Mutation in CALM1 Encoding Calmodulin in Familial Idiopathic Ventricular Fibrillation in Childhood and Adolescence

(2013) Roos F. Marsman et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

DNA targeting specificity of RNA-guided Cas9 nucleases

(2013) Patrick D Hsu et al.   NATURE BIOTECHNOLOGY

High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells

(2013) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

Genome engineering using the CRISPR-Cas9 system

(2013) F Ann Ran et al.   Nature Protocols

Modelling Human Channelopathies Using Induced Pluripotent Stem Cells: A Comprehensive Review

(2013) Martin Müller et al.   Stem Cells International

Mutations in Calmodulin Cause Ventricular Tachycardia and Sudden Cardiac Death

(2012) Mette Nyegaard et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Seeing the Forest through the Trees: towards a Unified View on Physiological Calcium Regulation of Voltage-Gated Sodium Channels

(2012) Filip Van Petegem et al.   BIOPHYSICAL JOURNAL

Maximum Diastolic Potential of Human Induced Pluripotent Stem Cell-Derived Cardiomyocytes Depends Critically on IKr

(2012) Michael Xavier Doss et al.   PLoS One

An Efficient Nonviral Method to Generate Integration-Free Human-Induced Pluripotent Stem Cells from Cord Blood and Peripheral Blood Cells

(2012) Keisuke Okita et al.   STEM CELLS

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

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

(2011) Masayuki Yazawa et al.   NATURE

KCNE4 Juxtamembrane Region Is Required for Interaction with Calmodulin and for Functional Suppression of KCNQ1

(2010) Erin J. Ciampa et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

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

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

Intermolecular tuning of calmodulin by target peptides and proteins: Differential effects on Ca2+ binding and implications for kinase activation

(2010) Olve B. Peersen et al.   PROTEIN SCIENCE

Human cardiovascular progenitor cells develop from a KDR+ embryonic-stem-cell-derived population

(2008) Lei Yang et al.   NATURE