Genome Editing in Induced Pluripotent Stem Cells using CRISPR/Cas9

CRISPR correction of the PRKAG2 gene mutation in the patient's induced pluripotent stem cell-derived cardiomyocytes eliminates electrophysiological and structural abnormalities

(2018) Ronen Ben Jehuda et al.   HEART RHYTHM

Molecular and functional resemblance of differentiated cells derived from isogenic human iPSCs and SCNT-derived ESCs

(2017) Ming-Tao Zhao et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Lack of MTTP Activity in Pluripotent Stem Cell-Derived Hepatocytes and Cardiomyocytes Abolishes apoB Secretion and Increases Cell Stress

(2017) Ying Liu et al.   Cell Reports

Proteasome impairment in neural cells derived from HMSN-P patient iPSCs

(2017) Nagahisa Murakami et al.   Molecular Brain

CRISPR/Cas9-mediated targeted gene correction in amyotrophic lateral sclerosis patient iPSCs

(2017) Lixia Wang et al.   Protein & Cell

Patient iPSC-Derived Neurons for Disease Modeling of Frontotemporal Dementia with Mutation in CHMP2B

(2017) Yu Zhang et al.   Stem Cell Reports

Reversal of Phenotypic Abnormalities by CRISPR/Cas9-Mediated Gene Correction in Huntington Disease Patient-Derived Induced Pluripotent Stem Cells

(2017) Xiaohong Xu et al.   Stem Cell Reports

A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells

(2016) Courtney S. Young et al.   Cell Stem Cell

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

Patient-Specific and Genome-Edited Induced Pluripotent Stem Cell–Derived Cardiomyocytes Elucidate Single-Cell Phenotype of Brugada Syndrome

(2016) Ping Liang et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Site-specific genome editing for correction of induced pluripotent stem cells derived from dominant dystrophic epidermolysis bullosa

(2016) Satoru Shinkuma et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

CRISPR-mediated genotypic and phenotypic correction of a chronic granulomatous disease mutation in human iPS cells

(2015) Rowan Flynn et al.   EXPERIMENTAL HEMATOLOGY

Functional abnormalities in iPSC-derived cardiomyocytes generated from CPVT1 and CPVT2 patients carrying ryanodine or calsequestrin mutations

(2015) Atara Novak et al.   JOURNAL OF CELLULAR AND MOLECULAR MEDICINE

Genetics of long-QT syndrome

(2015) Yukiko Nakano et al.   JOURNAL OF HUMAN GENETICS

Regulation of Postsynaptic Function by the Dementia-Related ESCRT-III Subunit CHMP2B

(2015) R. Chassefeyre et al.   JOURNAL OF NEUROSCIENCE

Efficient and Allele-Specific Genome Editing of Disease Loci in Human iPSCs

(2015) Cory Smith et al.   MOLECULAR THERAPY

Production of Gene-Corrected Adult Beta Globin Protein in Human Erythrocytes Differentiated from Patient iPSCs After Genome Editing of the Sickle Point Mutation

(2015) Xiaosong Huang et al.   STEM CELLS

Reversion of FMR1 Methylation and Silencing by Editing the Triplet Repeats in Fragile X iPSC-Derived Neurons

(2015) Chul-Yong Park et al.   Cell Reports

Long-Term Efficacy of Systemic Multiexon Skipping Targeting Dystrophin Exons 45–55 With a Cocktail of Vivo-Morpholinos in Mdx52 Mice

(2015) Yusuke Echigoya et al.   Molecular Therapy-Nucleic Acids

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

Seamless gene correction of β-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 andpiggyBac

(2014) Fei Xie et al.   GENOME RESEARCH

CRISPR-Cas systems for editing, regulating and targeting genomes

(2014) Jeffry D Sander et al.   NATURE BIOTECHNOLOGY

Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype

(2014) Hao Yin et al.   NATURE BIOTECHNOLOGY

Isolation of single-base genome-edited human iPS cells without antibiotic selection

(2014) Yuichiro Miyaoka et al.   NATURE METHODS

Cardiac Potassium Channel Subtypes: New Roles in Repolarization and Arrhythmia

(2014) Nicole Schmitt et al.   PHYSIOLOGICAL REVIEWS

One-Step Generation of Mice Carrying Mutations in Multiple Genes by CRISPR/Cas-Mediated Genome Engineering

(2013) Haoyi Wang et al.   CELL

Double Nicking by RNA-Guided CRISPR Cas9 for Enhanced Genome Editing Specificity

(2013) F. Ann Ran et al.   CELL

Correction of a Genetic Disease in Mouse via Use of CRISPR-Cas9

(2013) Yuxuan Wu et al.   Cell Stem Cell

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

Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease

(2013) Seung Woo Cho et al.   NATURE BIOTECHNOLOGY

High-throughput profiling of off-target DNA cleavage reveals RNA-programmed Cas9 nuclease specificity

(2013) Vikram Pattanayak et al.   NATURE BIOTECHNOLOGY

Genome engineering using the CRISPR-Cas9 system

(2013) F Ann Ran et al.   Nature Protocols

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

The TRK-Fused Gene Is Mutated in Hereditary Motor and Sensory Neuropathy with Proximal Dominant Involvement

(2012) Hiroyuki Ishiura et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Essential Features and Rational Design of CRISPR RNAs that Function with the Cas RAMP Module Complex to Cleave RNAs

(2012) Caryn R. Hale et al.   MOLECULAR CELL

A transcription activator-like effector toolbox for genome engineering

(2012) Neville E Sanjana et al.   Nature Protocols

Cas9-crRNA ribonucleoprotein complex mediates specific DNA cleavage for adaptive immunity in bacteria

(2012) G. Gasiunas et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

(2012) M. Jinek et al.   SCIENCE

Pathogenic Mutation of ALK2 Inhibits Induced Pluripotent Stem Cell Reprogramming and Maintenance: Mechanisms of Reprogramming and Strategy for Drug Identification

(2012) Makoto Hamasaki et al.   STEM CELLS

Induced Pluripotent Stem Cells from Ataxia-Telangiectasia Recapitulate the Cellular Phenotype

(2012) Sam Nayler et al.   Stem Cells Translational Medicine

Ultrastructure and molecular pathogenesis of epidermolysis bullosa

(2011) Satoru Shinkuma et al.   CLINICS IN DERMATOLOGY

Frontotemporal Dementia Caused by CHMP2B Mutations

(2011) A. M. Isaacs et al.   Current Alzheimer Research

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

HRS/EHRA Expert Consensus Statement on the State of Genetic Testing for the Channelopathies and Cardiomyopathies

(2011) Michael J. Ackerman et al.   HEART RHYTHM

Current Status of Pharmaceutical and Genetic Therapeutic Approaches to Treat DMD

(2011) Christophe Pichavant et al.   MOLECULAR THERAPY

CRISPR RNA maturation by trans-encoded small RNA and host factor RNase III

(2011) Elitza Deltcheva et al.   NATURE

Evolution and classification of the CRISPR–Cas systems

(2011) Kira S. Makarova et al.   NATURE REVIEWS MICROBIOLOGY

The Streptococcus thermophilus CRISPR/Cas system provides immunity in Escherichia coli

(2011) Rimantas Sapranauskas et al.   NUCLEIC ACIDS RESEARCH

Chromatin Structure and Gene Expression Programs of Human Embryonic and Induced Pluripotent Stem Cells

(2010) Matthew G. Guenther et al.   Cell Stem Cell

High risk of sudden death associated with a PRKAG2-related familial Wolff-Parkinson-White syndrome

(2010) Li-Ping Zhang et al.   JOURNAL OF ELECTROCARDIOLOGY

The diagnosis of young-onset dementia

(2010) Martin N Rossor et al.   LANCET NEUROLOGY

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

(2010) Marina Cavazzana-Calvo et al.   NATURE

Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease

(2010) Stefan Stein et al.   NATURE MEDICINE

Parkinson's Disease Patient-Derived Induced Pluripotent Stem Cells Free of Viral Reprogramming Factors

(2009) Frank Soldner et al.   CELL

Chronic Granulomatous Disease

(2009) Steven M. Holland   CLINICAL REVIEWS IN ALLERGY & IMMUNOLOGY

Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations

(2009) Annemieke Aartsma-Rus et al.   HUMAN MUTATION

Disorders of biopterin metabolism

(2009) Nicola Longo   JOURNAL OF INHERITED METABOLIC DISEASE

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management

(2009) Katharine Bushby et al.   LANCET NEUROLOGY

Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells

(2009) Ángel Raya et al.   NATURE

Generation of pluripotent stem cells from patients with type 1 diabetes

(2009) R. Maehr et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Breaking the Code of DNA Binding Specificity of TAL-Type III Effectors

(2009) J. Boch et al.   SCIENCE

Long QT Syndrome

(2008) Ilan Goldenberg et al.   CURRENT PROBLEMS IN CARDIOLOGY

The classification of inherited epidermolysis bullosa (EB): Report of the Third International Consensus Meeting on Diagnosis and Classification of EB

(2008) Jo-David Fine et al.   JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY

A protocol for removal of antibiotic resistance cassettes from human embryonic stem cells genetically modified by homologous recombination or transgenesis

(2008) Richard P Davis et al.   Nature Protocols

Small CRISPR RNAs Guide Antiviral Defense in Prokaryotes

(2008) S. J. J. Brouns et al.   SCIENCE