Genome Editing for the Study of Cardiovascular Diseases

CRISPR-Cas9 Targeting ofPCSK9in Human Hepatocytes In Vivo—Brief ReportSignificance

(2016) Xiao Wang et al.   ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY

ApoE knockout rabbits: A novel model for the study of human hyperlipidemia

(2016) Manabu Niimi et al.   ATHEROSCLEROSIS

Structure and Engineering of Francisella novicida Cas9

(2016) Hisato Hirano et al.   CELL

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

A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

(2016) Yang Yang et al.   NATURE BIOTECHNOLOGY

Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo

(2016) Hao Yin et al.   NATURE BIOTECHNOLOGY

A multifunctional AAV–CRISPR–Cas9 and its host response

(2016) Wei Leong Chew et al.   NATURE METHODS

Generation of heterozygous fibrillin-1 mutant cloned pigs from genome-edited foetal fibroblasts

(2016) Kazuhiro Umeyama et al.   Scientific Reports

Genome-Edited Human Pluripotent Stem Cell–Derived Macrophages as a Model of Reverse Cholesterol Transport

(2015) Rajat M. Gupta et al.   ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY

Human Disease Modeling Reveals Integrated Transcriptional and Epigenetic Mechanisms of NOTCH1 Haploinsufficiency

(2015) Christina V. Theodoris et al.   CELL

Cold-Inducible RNA-Binding Protein Regulates Cardiac Repolarization by Targeting Transient Outward Potassium Channels

(2015) J. Li et al.   CIRCULATION RESEARCH

Single epicardial cell transcriptome sequencing identifies Caveolin 1 as an essential factor in zebrafish heart regeneration

(2015) Jingli Cao et al.   DEVELOPMENT

Exacerbating Pressure Overload–Induced Cardiac HypertrophyNovelty and Significance

(2015) Xuehai Zhu et al.   HYPERTENSION

Engineered CRISPR-Cas9 nucleases with altered PAM specificities

(2015) Benjamin P. Kleinstiver et al.   NATURE

Genetic compensation induced by deleterious mutations but not gene knockdowns

(2015) Andrea Rossi et al.   NATURE

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

Increasing the efficiency of homology-directed repair for CRISPR-Cas9-induced precise gene editing in mammalian cells

(2015) Van Trung Chu et al.   NATURE BIOTECHNOLOGY

Therapeutic genome editing: prospects and challenges

(2015) David Benjamin Turitz Cox et al.   NATURE MEDICINE

Improved specificity of TALE-based genome editing using an expanded RVD repertoire

(2015) Jeffrey C Miller et al.   NATURE METHODS

Development of an intein-mediated split–Cas9 system for gene therapy

(2015) Dong-Jiunn Jeffery Truong et al.   NUCLEIC ACIDS RESEARCH

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

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

(2015) Ioannis Karakikes et al.   Nature Communications

Efficient generation of gene-modified pigs via injection of zygote with Cas9/sgRNA

(2015) Yong Wang et al.   Scientific Reports

An internal promoter underlies the difference in disease severity between N- and C-terminal truncation mutations of Titin in zebrafish

(2015) Jun Zou et al.   eLife

Efficient Multiple Genome Modifications Induced by the crRNAs, tracrRNA and Cas9 Protein Complex in Zebrafish

(2015) Hirohito Kotani et al.   PLoS One

klf2ash317 Mutant Zebrafish Do Not Recapitulate Morpholino-Induced Vascular and Haematopoietic Phenotypes

(2015) Peter Novodvorsky et al.   PLoS One

CRISPR-Cas9 Knockin Mice for Genome Editing and Cancer Modeling

(2014) Randall J. Platt et al.   CELL

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

(2014) Qiurong Ding et al.   CIRCULATION RESEARCH

Multiple genome modifications by the CRISPR/Cas9 system in zebrafish

(2014) Satoshi Ota et al.   GENES TO CELLS

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

Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

(2014) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

Dimeric CRISPR RNA-guided FokI nucleases for highly specific genome editing

(2014) Shengdar Q Tsai et al.   NATURE BIOTECHNOLOGY

Fusion of catalytically inactive Cas9 to FokI nuclease improves the specificity of genome modification

(2014) John P Guilinger 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

Gene Editing of CCR5 in Autologous CD4 T Cells of Persons Infected with HIV

(2014) Pablo Tebas et al.   NEW ENGLAND JOURNAL OF MEDICINE

Characterization of Dystrophin Deficient Rats: A New Model for Duchenne Muscular Dystrophy

(2014) Thibaut Larcher et al.   PLoS One

Mutation of Plekha7 attenuates salt-sensitive hypertension in the rat

(2014) B. T. Endres et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Recessive cardiac phenotypes in induced pluripotent stem cell models of Jervell and Lange-Nielsen syndrome: Disease mechanisms and pharmacological rescue

(2014) Miao Zhang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Efficient creation of an APOE knockout rabbit

(2014) Diana Ji et al.   TRANSGENIC RESEARCH

Effective gene targeting in rabbits using RNA-guided Cas9 nucleases

(2014) Dongshan Yang et al.   Journal of Molecular Cell Biology

Comparison of non-canonical PAMs for CRISPR/Cas9-mediated DNA cleavage in human cells

(2014) Yilan Zhang et al.   Scientific Reports

Enhanced homology-directed human genome engineering by controlled timing of CRISPR/Cas9 delivery

(2014) Steven Lin et al.   eLife

Genetic and Chemical Correction of Cholesterol Accumulation and Impaired Autophagy in Hepatic and Neural Cells Derived from Niemann-Pick Type C Patient-Specific iPS Cells

(2014) Dorothea Maetzel et al.   Stem Cell Reports

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

(2013) Haoyi Wang et al.   CELL

One-Step Generation of Mice Carrying Reporter and Conditional Alleles by CRISPR/Cas-Mediated Genome Engineering

(2013) Hui Yang et al.   CELL

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

(2013) F. Ann Ran et al.   CELL

Proprotein Convertase Subtilisin Kexin 9 (PCSK9) Inhibitors in the Treatment of Hypercholesterolemia and other Pathologies

(2013) Nabil G. Seidah   CURRENT PHARMACEUTICAL DESIGN

CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering

(2013) Prashant Mali et al.   NATURE BIOTECHNOLOGY

A library of TAL effector nucleases spanning the human genome

(2013) Yongsub Kim et al.   NATURE BIOTECHNOLOGY

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

(2013) Seung Woo Cho et al.   NATURE BIOTECHNOLOGY

Orthogonal Cas9 proteins for RNA-guided gene regulation and editing

(2013) Kevin M Esvelt et al.   NATURE METHODS

Efficient genome engineering in human pluripotent stem cells using Cas9 from Neisseria meningitidis

(2013) Z. Hou et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

(2013) Jianming Jiang et al.   SCIENCE

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 TALEN Genome-Editing System for Generating Human Stem Cell-Based Disease Models

(2012) Qiurong Ding et al.   Cell Stem Cell

Heart Disease and Stroke Statistics—2013 Update

(2012) Alan S. Go et al.   CIRCULATION

FLASH assembly of TALENs for high-throughput genome editing

(2012) Deepak Reyon et al.   NATURE BIOTECHNOLOGY

Targeted transcriptional activation of silent oct4 pluripotency gene by combining designer TALEs and inhibition of epigenetic modifiers

(2012) Sebastian Bultmann et al.   NUCLEIC ACIDS RESEARCH

Implications of the innate immune response to adenovirus and adenoviral vectors

(2011) Seth M Gregory et al.   Future Virology

Creation and Characterization of a Renin Knockout Rat

(2011) Carol Moreno et al.   HYPERTENSION

State-of-the-art gene-based therapies: the road ahead

(2011) Mark A. Kay   NATURE REVIEWS GENETICS

Efficient design and assembly of custom TALEN and other TAL effector-based constructs for DNA targeting

(2011) Tomas Cermak et al.   NUCLEIC ACIDS RESEARCH

A Proprotein Convertase Subtilisin-like/Kexin Type 9 (PCSK9) C-terminal Domain Antibody Antigen-binding Fragment Inhibits PCSK9 Internalization and Restores Low Density Lipoprotein Uptake

(2010) Yan G. Ni et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Engineering of Human Pluripotent Stem Cells by AAV-mediated Gene Targeting

(2010) Iram F Khan et al.   MOLECULAR THERAPY

Genome editing with engineered zinc finger nucleases

(2010) Fyodor D. Urnov et al.   NATURE REVIEWS GENETICS

Gene targeting in the rat: advances and opportunities

(2010) Howard J. Jacob et al.   TRENDS IN GENETICS

Antibody-mediated disruption of the interaction between PCSK9 and the low-density lipoprotein receptor

(2009) Christopher J. Duff et al.   BIOCHEMICAL JOURNAL

RNAi-based therapeutics-current status, challenges and prospects

(2009) Katrin Tiemann et al.   EMBO Molecular Medicine

Dissection of the Endogenous Cellular Pathways of PCSK9-induced Low Density Lipoprotein Receptor Degradation

(2009) Steve Poirier et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

The promises and pitfalls of RNA-interference-based therapeutics

(2009) Daniela Castanotto et al.   NATURE

Recognizing and avoiding siRNA off-target effects for target identification and therapeutic application

(2009) Aimee L. Jackson et al.   NATURE REVIEWS DRUG DISCOVERY

Efficient targeting of a SCID gene by an engineered single-chain homing endonuclease

(2009) Sylvestre Grizot et al.   NUCLEIC ACIDS RESEARCH

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

(2009) J. Boch et al.   SCIENCE

A Simple Cipher Governs DNA Recognition by TAL Effectors

(2009) M. J. Moscou et al.   SCIENCE

Molecular basis of PCSK9 function

(2008) Gilles Lambert et al.   ATHEROSCLEROSIS

PCSK9 binds to multiple receptors and can be functionally inhibited by an EGF-A peptide

(2008) LiXin Shan et al.   BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS

Unexpected failure rates for modular assembly of engineered zinc fingers

(2008) Cherie L Ramirez et al.   NATURE METHODS

Highly efficient transient gene expression and gene targeting in primate embryonic stem cells with helper-dependent adenoviral vectors

(2008) K. Suzuki et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA