CRISPR/Cas9 gene-editing strategies in cardiovascular cells

In vivo genome and base editing of a human PCSK9 knock-in hypercholesterolemic mouse model

(2019) Alba Carreras et al.   BMC BIOLOGY

A Premature Termination Codon Mutation in MYBPC3 Causes Hypertrophic Cardiomyopathy via Chronic Activation of Nonsense-Mediated Decay

(2019) Timon Seeger et al.   CIRCULATION

The Future of Gene Editing — Toward Scientific and Social Consensus

(2019) Lisa Rosenbaum   NEW ENGLAND JOURNAL OF MEDICINE

After the Storm — A Responsible Path for Genome Editing

(2019) George Q. Daley et al.   NEW ENGLAND JOURNAL OF MEDICINE

Methodologies for Improving HDR Efficiency

(2019) Mingjie Liu et al.   Frontiers in Genetics

Knockout rat models mimicking human atherosclerosis created by Cpf1-mediated gene targeting

(2019) Jong Geol Lee et al.   Scientific Reports

Identification of preexisting adaptive immunity to Cas9 proteins in humans

(2019) Carsten T. Charlesworth et al.   NATURE MEDICINE

Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy

(2019) Christopher E. Nelson et al.   NATURE MEDICINE

Engineered CRISPR–Cas12a variants with increased activities and improved targeting ranges for gene, epigenetic and base editing

(2019) Benjamin P. Kleinstiver et al.   NATURE BIOTECHNOLOGY

Patient-independent human induced pluripotent stem cell model: A new tool for rapid determination of genetic variant pathogenicity in long QT syndrome

(2019) Nikhil V. Chavali et al.   HEART RHYTHM

Reduced Blood Lipid Levels With In Vivo CRISPR-Cas9 Base Editing of ANGPTL3

(2018) Alexandra C. Chadwick et al.   CIRCULATION

A novel rabbit model of Duchenne muscular dystrophy generated by CRISPR/Cas9

(2018) Tingting Sui et al.   Disease Models & Mechanisms

Delivering CRISPR: a review of the challenges and approaches

(2018) Christopher A. Lino et al.   DRUG DELIVERY

Unexpected heterogeneity derived from Cas9 ribonucleoprotein-introduced clonal cells at the HPRT1 locus

(2018) Tetsushi Sakuma et al.   GENES TO CELLS

Haemophilia gene therapy: From trailblazer to gamechanger

(2018) H. Evens et al.   HAEMOPHILIA

Establishment of a PRKAG2 cardiac syndrome disease model and mechanism study using human induced pluripotent stem cells

(2018) Yongkun Zhan et al.   JOURNAL OF MOLECULAR AND CELLULAR CARDIOLOGY

Genome Editing of Induced Pluripotent Stem Cells to Decipher Cardiac Channelopathy Variant

(2018) Priyanka Garg et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

The Evolving Future of PCSK9 Inhibitors

(2018) Robert S. Rosenson et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Efficient In Vivo Liver-Directed Gene Editing Using CRISPR/Cas9

(2018) Kshitiz Singh et al.   MOLECULAR THERAPY

p53 inhibits CRISPR–Cas9 engineering in human pluripotent stem cells

(2018) Robert J. Ihry et al.   NATURE MEDICINE

CRISPR–Cas9 genome editing induces a p53-mediated DNA damage response

(2018) Emma Haapaniemi et al.   NATURE MEDICINE

Integrated design, execution, and analysis of arrayed and pooled CRISPR genome-editing experiments

(2018) Matthew C Canver et al.   Nature Protocols

Strategies for In Vivo Genome Editing in Nondividing Cells

(2018) Fatemeharefeh Nami et al.   TRENDS IN BIOTECHNOLOGY

A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing

(2018) Jonathan D. Finn et al.   Cell Reports

In Vivo Ryr 2 Editing Corrects Catecholaminergic Polymorphic Ventricular Tachycardia

(2018) Xiaolu Pan et al.   CIRCULATION RESEARCH

Correction of the Marfan Syndrome Pathogenic FBN1 Mutation by Base Editing in Human Cells and Heterozygous Embryos

(2018) Yanting Zeng et al.   MOLECULAR THERAPY

In vivo CRISPR editing with no detectable genome-wide off-target mutations

(2018) Pinar Akcakaya et al.   NATURE

Genome Editing of Pigs for Agriculture and Biomedicine

(2018) Huaqiang Yang et al.   Frontiers in Genetics

High prevalence of Streptococcus pyogenes Cas9-reactive T cells within the adult human population

(2018) Dimitrios L. Wagner et al.   NATURE MEDICINE

In utero CRISPR-mediated therapeutic editing of metabolic genes

(2018) Avery C. Rossidis et al.   NATURE MEDICINE

Genome editing in primary cells and in vivo using viral-derived Nanoblades loaded with Cas9-sgRNA ribonucleoproteins

(2018) Philippe E. Mangeot et al.   Nature Communications

Cre-dependent Cas9-expressing pigs enable efficient in vivo genome editing

(2017) Kepin Wang et al.   GENOME RESEARCH

Genome Editing

(2017) Kiran Musunuru   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Optimizing Cardiac Delivery of Modified mRNA

(2017) Nishat Sultana et al.   MOLECULAR THERAPY

Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage

(2017) Nicole M. Gaudelli et al.   NATURE

Correction of a pathogenic gene mutation in human embryos

(2017) Hong Ma et al.   NATURE

The control of DNA repair by the cell cycle

(2017) Nicole Hustedt et al.   NATURE CELL BIOLOGY

CIRCLE-seq: a highly sensitive in vitro screen for genome-wide CRISPR–Cas9 nuclease off-targets

(2017) Shengdar Q Tsai et al.   NATURE METHODS

Gene therapy: Human genome editing in heart disease

(2017) Gregory B. Lim   NATURE REVIEWS GENETICS

Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy

(2017) Leonela Amoasii et al.   Science Translational Medicine

In Silico Meets In Vivo : Towards Computational CRISPR-Based sgRNA Design

(2017) Guo-hui Chuai et al.   TRENDS IN BIOTECHNOLOGY

In vivo genome editing with a small Cas9 orthologue derived from Campylobacter jejuni

(2017) Eunji Kim et al.   Nature Communications

Assembly of CRISPR ribonucleoproteins with biotinylated oligonucleotides via an RNA aptamer for precise gene editing

(2017) Jared Carlson-Stevermer et al.   Nature Communications

Efficient DNA-free genome editing of bread wheat using CRISPR/Cas9 ribonucleoprotein complexes

(2017) Zhen Liang et al.   Nature Communications

Optimization of the production of knock-in alleles by CRISPR/Cas9 microinjection into the mouse zygote

(2017) Aurélien Raveux et al.   Scientific Reports

Somatic genome editing with CRISPR/Cas9 generates and corrects a metabolic disease

(2017) Kelsey E. Jarrett et al.   Scientific Reports

Genome editing with CRISPR/Cas9 in postnatal mice corrects PRKAG2 cardiac syndrome

(2016) Chang Xie et al.   CELL RESEARCH

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

Gene therapy for the treatment of heart failure: promise postponed

(2016) Jean-Sebastien Hulot et al.   EUROPEAN HEART JOURNAL

Adeno-Associated Virus Gene Therapy for Liver Disease

(2016) Lisa M. Kattenhorn et al.   HUMAN GENE THERAPY

Translation of Human-Induced Pluripotent Stem Cells

(2016) Nazish Sayed et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage

(2016) Alexis C. Komor et al.   NATURE

High-fidelity CRISPR–Cas9 nucleases with no detectable genome-wide off-target effects

(2016) Benjamin P. Kleinstiver et al.   NATURE

Enhancing homology-directed genome editing by catalytically active and inactive CRISPR-Cas9 using asymmetric donor DNA

(2016) Christopher D Richardson et al.   NATURE BIOTECHNOLOGY

Multiplex gene editing by CRISPR–Cpf1 using a single crRNA array

(2016) Bernd Zetsche et al.   NATURE BIOTECHNOLOGY

Induced pluripotent stem cell technology: a decade of progress

(2016) Yanhong Shi et al.   NATURE REVIEWS DRUG DISCOVERY

Human Induced Pluripotent Stem Cells as a Platform for Personalized and Precision Cardiovascular Medicine

(2016) Elena Matsa et al.   PHYSIOLOGICAL REVIEWS

Concordant but Varied Phenotypes among Duchenne Muscular Dystrophy Patient-Specific Myoblasts Derived using a Human iPSC-Based Model

(2016) In Young Choi et al.   Cell Reports

Nucleosomes impede Cas9 access to DNA in vivo and in vitro

(2016) Max A Horlbeck et al.   eLife

Phospholamban Ablation Using CRISPR/Cas9 System Improves Mortality in a Murine Heart Failure Model

(2016) Manami Kaneko et al.   PLoS One

Nucleosomes Inhibit Cas9 Endonuclease Activity in Vitro

(2015) John M. Hinz et al.   BIOCHEMISTRY

Multiplex Genome-Edited T-cell Manufacturing Platform for “Off-the-Shelf” Adoptive T-cell Immunotherapies

(2015) Laurent Poirot et al.   CANCER RESEARCH

Gene therapy for cardiovascular disease: advances in vector development, targeting, and delivery for clinical translation

(2015) Melvin Y. Rincon et al.   CARDIOVASCULAR RESEARCH

Cpf1 Is a Single RNA-Guided Endonuclease of a Class 2 CRISPR-Cas System

(2015) Bernd Zetsche et al.   CELL

How specific is CRISPR/Cas9 really?

(2015) Henriette O’Geen et al.   CURRENT OPINION IN CHEMICAL BIOLOGY

Engineered CRISPR-Cas9 nucleases with altered PAM specificities

(2015) Benjamin P. Kleinstiver et al.   NATURE

Digenome-seq: genome-wide profiling of CRISPR-Cas9 off-target effects in human cells

(2015) Daesik Kim et al.   NATURE METHODS

An updated evolutionary classification of CRISPR–Cas systems

(2015) Kira S. Makarova et al.   NATURE REVIEWS MICROBIOLOGY

A mouse model for adult cardiac-specific gene deletion with CRISPR/Cas9

(2015) Kelli J. Carroll et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Titin mutations in iPS cells define sarcomere insufficiency as a cause of dilated cardiomyopathy

(2015) J. T. Hinson et al.   SCIENCE

Rationally engineered Cas9 nucleases with improved specificity

(2015) I. M. Slaymaker et al.   SCIENCE

Dynamics of CRISPR-Cas9 genome interrogation in living cells

(2015) Spencer C. Knight et al.   SCIENCE

Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy

(2015) C. Long et al.   SCIENCE

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

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

(2015) Ioannis Karakikes et al.   Nature Communications

Genome-Scale CRISPR-Mediated Control of Gene Repression and Activation

(2014) Luke A. Gilbert et al.   CELL

Development and Applications of CRISPR-Cas9 for Genome Engineering

(2014) Patrick D. Hsu et al.   CELL

Permanent Alteration of PCSK9 With In Vivo CRISPR-Cas9 Genome Editing

(2014) Qiurong Ding et al.   CIRCULATION RESEARCH

Somatic mosaicism and allele complexity induced by CRISPR/Cas9 RNA injections in mouse zygotes

(2014) Shuo-Ting Yen et al.   DEVELOPMENTAL BIOLOGY

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

Structural basis of PAM-dependent target DNA recognition by the Cas9 endonuclease

(2014) Carolin Anders et al.   NATURE

Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

(2014) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

CRISPR-Cas systems for editing, regulating and targeting genomes

(2014) Jeffry D Sander et al.   NATURE BIOTECHNOLOGY

GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases

(2014) Shengdar Q Tsai et al.   NATURE BIOTECHNOLOGY

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

(2014) Gang Wang et al.   NATURE MEDICINE

Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA

(2014) C. Long et al.   SCIENCE

The new frontier of genome engineering with CRISPR-Cas9

(2014) J. A. Doudna et al.   SCIENCE

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

(2013) Haoyi Wang et al.   CELL

Repurposing CRISPR as an RNA-Guided Platform for Sequence-Specific Control of Gene Expression

(2013) Lei S. Qi et al.   CELL

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

(2013) F. Ann Ran et al.   CELL

Multiplexed activation of endogenous genes by CRISPR-on, an RNA-guided transcriptional activator system

(2013) Albert W Cheng et al.   CELL RESEARCH

DNA targeting specificity of RNA-guided Cas9 nucleases

(2013) Patrick D Hsu 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

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

(2013) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

Cas9 as a versatile tool for engineering biology

(2013) Prashant Mali et al.   NATURE METHODS

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

RNA-programmed genome editing in human cells

(2013) Martin Jinek et al.   eLife

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

(2012) M. Jinek et al.   SCIENCE

Genome Engineering With Zinc-Finger Nucleases

(2011) Dana Carroll   GENETICS

Hotspots of aberrant epigenomic reprogramming in human induced pluripotent stem cells

(2011) Ryan Lister et al.   NATURE

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

(2011) Elitza Deltcheva et al.   NATURE

TAL effectors: finding plant genes for disease and defense

(2010) Adam J Bogdanove et al.   CURRENT OPINION IN PLANT BIOLOGY

Targeting DNA Double-Strand Breaks with TAL Effector Nucleases

(2010) M. Christian et al.   GENETICS

Comparison of nonhomologous end joining and homologous recombination in human cells

(2008) Z MAO et al.   DNA REPAIR