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Title
CRISPR Modeling and Correction of Cardiovascular Disease
Authors
Keywords
-
Journal
CIRCULATION RESEARCH
Volume 130, Issue 12, Pages 1827-1850
Publisher
Ovid Technologies (Wolters Kluwer Health)
Online
2022-06-10
DOI
10.1161/circresaha.122.320496
References
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Note: Only part of the references are listed.- Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins
- (2022) Samagya Banskota et al. CELL
- The use of new CRISPR tools in cardiovascular research and medicine
- (2022) Masataka Nishiga et al. Nature Reviews Cardiology
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- (2022) Kalina Paunovska et al. NATURE REVIEWS GENETICS
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- (2022) Xianming Zhang et al. Cell Reports
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- (2022) Hiroyuki Inoue et al. Stem Cell Reports
- Endothelial Retargeting of AAV9 In Vivo
- (2022) Tarik Bozoglu et al. Advanced Science
- Efficient In Vivo Homology-Directed Repair Within Cardiomyocytes
- (2022) Yanjiang Zheng et al. CIRCULATION
- Genetic Variation in Enhancers Modifies Cardiomyopathy Gene Expression and Progression
- (2021) Anthony M. Gacita et al. CIRCULATION
- In vivo base editing rescues Hutchinson–Gilford progeria syndrome in mice
- (2021) Luke W. Koblan et al. NATURE
- Identifying genome-wide off-target sites of CRISPR RNA–guided nucleases and deaminases with Digenome-seq
- (2021) Daesik Kim et al. Nature Protocols
- Lipid nanoparticle-mediated codelivery of Cas9 mRNA and single-guide RNA achieves liver-specific in vivo genome editing of Angptl3
- (2021) Min Qiu et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Adenine Base Editor Ribonucleoproteins Delivered by Lentivirus-Like Particles Show High On-Target Base Editing and Undetectable RNA Off-Target Activities
- (2021) Pin Lyu et al. CRISPR Journal
- In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates
- (2021) Kiran Musunuru et al. NATURE
- In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels
- (2021) Tanja Rothgangl et al. NATURE BIOTECHNOLOGY
- Discovery and engineering of small SlugCas9 with broad targeting range and high specificity and activity
- (2021) Ziying Hu et al. NUCLEIC ACIDS RESEARCH
- Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice
- (2021) Pengpeng Liu et al. Nature Communications
- Nanoparticle Delivery of CRISPR/Cas9 for Genome Editing
- (2021) Li Duan et al. Frontiers in Genetics
- Precise correction of Duchenne muscular dystrophy exon deletion mutations by base and prime editing
- (2021) F. Chemello et al. Science Advances
- Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery across species
- (2021) Mohammadsharif Tabebordbar et al. CELL
- Efficient Correction of a Hypertrophic Cardiomyopathy Mutation by ABEmax-NG
- (2021) Shuhong Ma et al. CIRCULATION RESEARCH
- Cardiac Myoediting Attenuates Cardiac Abnormalities in Human and Mouse Models of Duchenne Muscular Dystrophy
- (2021) Ayhan Atmanli et al. CIRCULATION RESEARCH
- Loss of crossbridge inhibition drives pathological cardiac hypertrophy in patients harboring the TPM1 E192K mutation
- (2021) Lorenzo R. Sewanan et al. JOURNAL OF GENERAL PHYSIOLOGY
- Versatile and efficient in vivo genome editing with compact Streptococcus pasteurianus Cas9
- (2021) Zhiquan Liu et al. MOLECULAR THERAPY
- Genomic frontiers in congenital heart disease
- (2021) Sarah U. Morton et al. Nature Reviews Cardiology
- Gene therapy knockdown of Hippo signaling induces cardiomyocyte renewal in pigs after myocardial infarction
- (2021) Shijie Liu et al. Science Translational Medicine
- Improved CRISPR genome editing using small highly active and specific engineered RNA-guided nucleases
- (2021) Moritz J. Schmidt et al. Nature Communications
- In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease
- (2021) Sourav K. Bose et al. Nature Communications
- Massively parallel in vivo CRISPR screening identifies RNF20/40 as epigenetic regulators of cardiomyocyte maturation
- (2021) Nathan J. VanDusen et al. Nature Communications
- Efficient precise in vivo base editing in adult dystrophic mice
- (2021) Li Xu et al. Nature Communications
- Targeted delivery of CRISPR-Cas9 and transgenes enables complex immune cell engineering
- (2021) Jennifer R. Hamilton et al. Cell Reports
- A consolidated AAV system for single-cut CRISPR correction of a common Duchenne muscular dystrophy mutation
- (2021) Yu Zhang et al. Molecular Therapy-Methods & Clinical Development
- Application of prime editing to the correction of mutations and phenotypes in adult mice with liver and eye diseases
- (2021) Hyewon Jang et al. Nature Biomedical Engineering
- Understanding the molecular basis of cardiomyopathy
- (2021) Marie-Louise Bang et al. AMERICAN JOURNAL OF PHYSIOLOGY-HEART AND CIRCULATORY PHYSIOLOGY
- Enhanced prime editing systems by manipulating cellular determinants of editing outcomes
- (2021) Peter J. Chen et al. CELL
- CRISPR/Cas correction of muscular dystrophies
- (2021) Yu Zhang et al. EXPERIMENTAL CELL RESEARCH
- Engineered pegRNAs improve prime editing efficiency
- (2021) James W. Nelson et al. NATURE BIOTECHNOLOGY
- Defining genome-wide CRISPR–Cas genome-editing nuclease activity with GUIDE-seq
- (2021) Nikolay L. Malinin et al. Nature Protocols
- Generation and genetic repair of two human induced pluripotent cell lines from patients with Epidermolysis Bullosa simplex and dilated cardiomyopathy associated with a heterozygous mutation in the translation initiation codon of KLHL24
- (2021) Veronika Ramovs et al. Stem Cell Research
- Low immunogenicity of LNP allows repeated administrations of CRISPR-Cas9 mRNA into skeletal muscle in mice
- (2021) Eriya Kenjo et al. Nature Communications
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- (2021) Raed Ibraheim et al. Nature Communications
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- (2021) Chady H. Hakim et al. Nature Communications
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- (2021) Bilge E Öztürk et al. eLife
- Adeno-Associated Viruses (AAV) and Host Immunity – A Race Between the Hare and the Hedgehog
- (2021) Kleopatra Rapti et al. Frontiers in Immunology
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- (2021) Ibrahim El‐Battrawy et al. Clinical and Translational Medicine
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- (2020) Masataka Nishiga et al. ADVANCED DRUG DELIVERY REVIEWS
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- (2020) James M. Wilson et al. HUMAN GENE THERAPY
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- (2020) Anthony M. Pettinato et al. CIRCULATION
- Gene Therapy With the DWORF Micropeptide Attenuates Cardiomyopathy in Mice
- (2020) Catherine A. Makarewich et al. CIRCULATION RESEARCH
- Epidemiology of the inherited cardiomyopathies
- (2020) William J. McKenna et al. Nature Reviews Cardiology
- Base editing: advances and therapeutic opportunities
- (2020) Elizabeth M. Porto et al. NATURE REVIEWS DRUG DISCOVERY
- Prime editing for functional repair in patient-derived disease models
- (2020) Imre F. Schene et al. Nature Communications
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- (2020) Yasuaki Kohama et al. Scientific Reports
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- (2020) Zachary C. Elmore et al. JCI Insight
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- (2020) Irfan S. Kathiriya et al. DEVELOPMENTAL CELL
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- (2020) Michael J. Mitchell et al. NATURE REVIEWS DRUG DISCOVERY
- In Vivo Gene Editing of Muscle Stem Cells with Adeno-Associated Viral Vectors in a Mouse Model of Duchenne Muscular Dystrophy
- (2020) Jennifer B. Kwon et al. Molecular Therapy-Methods & Clinical Development
- A Premature Termination Codon Mutation in MYBPC3 Causes Hypertrophic Cardiomyopathy via Chronic Activation of Nonsense-Mediated Decay
- (2019) Timon Seeger et al. CIRCULATION
- CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells
- (2019) Yi-Li Min et al. Science Advances
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- (2019) Jianguo Zhao et al. National Science Review
- Cytosine base editor generates substantial off-target single-nucleotide variants in mouse embryos
- (2019) Erwei Zuo et al. SCIENCE
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- (2019) Shuai Jin et al. SCIENCE
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- (2019) R. Grant Rowe et al. NATURE REVIEWS GENETICS
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- (2019) Dan Wang et al. NATURE REVIEWS DRUG DISCOVERY
- 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
- Development of a CRISPR/Cas9-based therapy for Hutchinson–Gilford progeria syndrome
- (2019) Olaya Santiago-Fernández et al. NATURE MEDICINE
- Single-dose CRISPR–Cas9 therapy extends lifespan of mice with Hutchinson–Gilford progeria syndrome
- (2019) Ergin Beyret et al. NATURE MEDICINE
- Protease-Activatable Adeno-Associated Virus Vector for Gene Delivery to Damaged Heart Tissue
- (2019) Caitlin M. Guenther et al. MOLECULAR THERAPY
- Combinatorial interactions of genetic variants in human cardiomyopathy
- (2019) Dekker C. Deacon et al. Nature Biomedical Engineering
- Life-Long AAV-Mediated CRISPR Genome Editing in Dystrophic Heart Improves Cardiomyopathy without Causing Serious Lesions in mdx Mice
- (2019) Li Xu et al. MOLECULAR THERAPY
- Transcriptome-wide off-target RNA editing induced by CRISPR-guided DNA base editors
- (2019) Julian Grünewald et al. NATURE
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- (2019) Sukanya Iyer et al. NATURE
- The next generation of CRISPR–Cas technologies and applications
- (2019) Adrian Pickar-Oliver et al. NATURE REVIEWS MOLECULAR CELL BIOLOGY
- Therapeutic developments for Duchenne muscular dystrophy
- (2019) Ingrid E. C. Verhaart et al. Nature Reviews Neurology
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- (2019) Mareike D Hoffmann et al. NUCLEIC ACIDS RESEARCH
- In vivo genome editing rescues photoreceptor degeneration via a Cas9/RecA-mediated homology-directed repair pathway
- (2019) Yuan Cai et al. Science Advances
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- (2019) Holly A. Rees et al. Science Advances
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- (2019) Fabrice Jaffré et al. CIRCULATION
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- (2019) Michael E. Nance et al. MOLECULAR THERAPY
- Off-target RNA mutation induced by DNA base editing and its elimination by mutagenesis
- (2019) Changyang Zhou et al. NATURE
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- (2019) Pin Lyu et al. NUCLEIC ACIDS RESEARCH
- Oligogenic inheritance of a human heart disease involving a genetic modifier
- (2019) Casey A. Gifford et al. SCIENCE
- Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here?
- (2019) Allison M. Keeler et al. Annual Review of Virology
- A biodegradable nanocapsule delivers a Cas9 ribonucleoprotein complex for in vivo genome editing
- (2019) Guojun Chen et al. Nature Nanotechnology
- Tissue-restricted Genome Editing in vivo Specified by MicroRNA-repressible Anti-CRISPR Proteins
- (2019) Jooyoung Lee et al. RNA
- CRISPR-Mediated Activation of Endogenous Gene Expression in the Postnatal Heart
- (2019) Eric Schoger et al. CIRCULATION RESEARCH
- Search-and-replace genome editing without double-strand breaks or donor DNA
- (2019) Andrew V. Anzalone et al. NATURE
- Optimized CRISPR guide RNA design for two high-fidelity Cas9 variants by deep learning
- (2019) Daqi Wang et al. Nature Communications
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- (2019) Erica McGrath et al. Nature Communications
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- (2019) Sheng Tong et al. Nature Reviews Materials
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- (2019) Dong Hyun Jo et al. Science Advances
- Advances in genome editing through control of DNA repair pathways
- (2019) Charles D. Yeh et al. NATURE CELL BIOLOGY
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- (2018) Kelsey E. Jarrett et al. ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY
- The Biology of CRISPR-Cas: Backward and Forward
- (2018) Frank Hille et al. CELL
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- (2018) Ning Ma et al. CIRCULATION
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- (2018) Priyanka Garg et al. JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY
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- (2018) Taeyoung Koo et al. MOLECULAR THERAPY
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- (2018) Antonio Casini et al. NATURE BIOTECHNOLOGY
- Myoediting: Toward Prevention of Muscular Dystrophy by Therapeutic Genome Editing
- (2018) Yu Zhang et al. PHYSIOLOGICAL REVIEWS
- Gene therapy comes of age
- (2018) Cynthia E. Dunbar et al. SCIENCE
- Incorporation of bridged nucleic acids into CRISPR RNAs improves Cas9 endonuclease specificity
- (2018) Christopher R. Cromwell et al. Nature Communications
- The CRISPR tool kit for genome editing and beyond
- (2018) Mazhar Adli Nature Communications
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- (2018) J. Montag et al. Scientific Reports
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- (2018) Jonathan D. Finn et al. Cell Reports
- Prevalence of Pre-existing Antibodies to CRISPR-Associated Nuclease Cas9 in the USA Population
- (2018) Vijaya L. Simhadri et al. Molecular Therapy-Methods & Clinical Development
- Correction of diverse muscular dystrophy mutations in human engineered heart muscle by single-site genome editing
- (2018) Chengzu Long et al. Science Advances
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- (2018) Xiaolu Pan et al. CIRCULATION RESEARCH
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- (2018) Yanting Zeng et al. MOLECULAR THERAPY
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- (2018) Johnny H. Hu et al. NATURE
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- (2018) Leonela Amoasii et al. SCIENCE
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- (2018) Gavin J. Knott et al. SCIENCE
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- (2018) Yuxuan Guo et al. Nature Communications
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- (2018) Eloisa Arbustini et al. JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY
- A Compact, High-Accuracy Cas9 with a Dinucleotide PAM for In Vivo Genome Editing
- (2018) Alireza Edraki et al. MOLECULAR CELL
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- (2018) Anob M. Chakrabarti et al. MOLECULAR CELL
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- (2018) Dimitrios L. Wagner et al. NATURE MEDICINE
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- (2018) Lukas Villiger et al. NATURE MEDICINE
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- (2018) Cicera R. Lazzarotto et al. Nature Protocols
- Base editing: precision chemistry on the genome and transcriptome of living cells
- (2018) Holly A. Rees et al. NATURE REVIEWS GENETICS
- 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
- A Self-Deleting AAV-CRISPR System for In Vivo Genome Editing
- (2018) Ang Li et al. Molecular Therapy-Methods & Clinical Development
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- (2017) Nicholas J. Silvestri et al. MUSCLE & NERVE
- Enhanced proofreading governs CRISPR–Cas9 targeting accuracy
- (2017) Janice S. Chen et al. NATURE
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- (2017) Nicole M. Gaudelli et al. NATURE
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- (2017) Hong Ma et al. NATURE
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- (2017) Sergey Shmakov et al. NATURE REVIEWS MICROBIOLOGY
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- (2017) Jun Nishiyama et al. NEURON
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- (2017) Eunji Kim et al. Nature Communications
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- (2017) Gianluca Petris et al. Nature Communications
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- (2017) Lei Huang et al. Oncotarget
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- (2017) Yuya Kobayashi et al. Genome Medicine
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- (2017) Takamaru Ishizu et al. Scientific Reports
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- (2016) Daria Wojtal et al. AMERICAN JOURNAL OF HUMAN GENETICS
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- (2016) Chang Xie et al. CELL RESEARCH
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- (2016) Courtney S. Young et al. Cell Stem Cell
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- (2016) Natalia Tapia et al. Cell Stem Cell
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- (2016) J G Choi et al. GENE THERAPY
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- (2016) Megan van Overbeek et al. MOLECULAR CELL
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- (2016) Ciaran M Lee et al. MOLECULAR THERAPY
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- (2016) Keiichiro Suzuki et al. NATURE
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- (2016) Alexis C. Komor et al. NATURE
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- (2016) Benjamin P. Kleinstiver et al. NATURE
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- (2016) Kazuki Kodo et al. NATURE CELL BIOLOGY
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- (2016) Jenny A. Greig et al. VACCINE
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- (2016) David Cano-Rodriguez et al. Nature Communications
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- (2016) Kazuhiro Umeyama et al. Scientific Reports
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- (2015) Sue Richards et al. GENETICS IN MEDICINE
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- (2015) Yongchang Chen et al. HUMAN MOLECULAR GENETICS
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- (2015) C Bruce A Whitelaw et al. JOURNAL OF PATHOLOGY
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- (2015) You Li et al. NATURE
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- (2015) Silvana Konermann et al. NATURE
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- (2015) F. Ann Ran et al. NATURE
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- (2015) Isaac B Hilton et al. NATURE BIOTECHNOLOGY
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- (2015) Nicola A Kearns et al. NATURE METHODS
- Highly efficient Cas9-mediated transcriptional programming
- (2015) Alejandro Chavez et al. NATURE METHODS
- 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
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- (2015) I. M. Slaymaker et al. SCIENCE
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- (2015) C. Long et al. SCIENCE
- In vivo gene editing in dystrophic mouse muscle and muscle stem cells
- (2015) M. Tabebordbar et al. SCIENCE
- Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy
- (2015) David G. Ousterout et al. Nature Communications
- Efficient generation of gene-modified pigs via injection of zygote with Cas9/sgRNA
- (2015) Yong Wang et al. Scientific Reports
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- (2015) Roberto Calcedo et al. Human Gene Therapy Methods
- Crystal Structure of Cas9 in Complex with Guide RNA and Target DNA
- (2014) Hiroshi Nishimasu et al. CELL
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- (2014) Samuel H. Sternberg et al. NATURE
- Improving CRISPR-Cas nuclease specificity using truncated guide RNAs
- (2014) Yanfang Fu 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
- Nature's recipe for splitting inteins
- (2014) A. S. Aranko et al. PROTEIN ENGINEERING DESIGN & SELECTION
- Utilizing Multiple in Silico Analyses to Identify Putative Causal SCN5A Variants in Brugada Syndrome
- (2014) Jyh-Ming Jimmy Juang et al. Scientific Reports
- Enhancing gene delivery of adeno-associated viruses by cell-permeable peptides
- (2014) Yarong Liu et al. Molecular Therapy-Methods & Clinical Development
- 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
- 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
- CRISPR-Mediated Modular RNA-Guided Regulation of Transcription in Eukaryotes
- (2013) Luke A. Gilbert et al. CELL
- Mechanistic Insights into the Enhancement of Adeno-Associated Virus Transduction by Proteasome Inhibitors
- (2013) Angela M. Mitchell et al. JOURNAL OF VIROLOGY
- Noonan syndrome
- (2013) Amy E Roberts et al. LANCET
- High-throughput profiling of off-target DNA cleavage reveals RNA-programmed Cas9 nuclease specificity
- (2013) Vikram Pattanayak et al. NATURE BIOTECHNOLOGY
- RNA-guided gene activation by CRISPR-Cas9–based transcription factors
- (2013) Pablo Perez-Pinera et al. NATURE METHODS
- CRISPR RNA–guided activation of endogenous human genes
- (2013) Morgan L Maeder et al. NATURE METHODS
- 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
- Cardiac-Specific Inducible and Conditional Gene Targeting in Mice
- (2012) Thomas Doetschman et al. CIRCULATION RESEARCH
- Genetics of Hypertrophic Cardiomyopathy After 20 Years
- (2012) Barry J. Maron et al. JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY
- A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity
- (2012) M. Jinek et al. SCIENCE
- Genome-wide Studies of Copy Number Variation and Exome Sequencing Identify Rare Variants in BAG3 as a Cause of Dilated Cardiomyopathy
- (2011) Nadine Norton et al. AMERICAN JOURNAL OF HUMAN GENETICS
- Generation of PPARγ mono-allelic knockout pigs via zinc-finger nucleases and nuclear transfer cloning
- (2011) Dongshan Yang et al. CELL RESEARCH
- A genome-wide association study identifies two loci associated with heart failure due to dilated cardiomyopathy
- (2011) Eric Villard et al. EUROPEAN HEART JOURNAL
- Impact of Pre-Existing Immunity on Gene Transfer to Nonhuman Primate Liver with Adeno-Associated Virus 8 Vectors
- (2011) Lili Wang et al. HUMAN GENE THERAPY
- Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector
- (2011) Dawn E Bowles et al. MOLECULAR THERAPY
- Evolution and classification of the CRISPR–Cas systems
- (2011) Kira S. Makarova et al. NATURE REVIEWS MICROBIOLOGY
- Liver-specific microRNA-122 target sequences incorporated in AAV vectors efficiently inhibits transgene expression in the liver
- (2010) C Qiao et al. GENE THERAPY
- MicroRNA-regulated, Systemically Delivered rAAV9: A Step Closer to CNS-restricted Transgene Expression
- (2010) Jun Xie et al. MOLECULAR THERAPY
- A method and server for predicting damaging missense mutations
- (2010) Ivan A Adzhubei et al. NATURE METHODS
- Nonsense-Mediated mRNA Decay and Ubiquitin–Proteasome System Regulate Cardiac Myosin-Binding Protein C Mutant Levels in Cardiomyopathic Mice
- (2009) Nicolas Vignier et al. CIRCULATION RESEARCH
- Proteasome Inhibitors Decrease AAV2 Capsid derived Peptide Epitope Presentation on MHC Class I Following Transduction
- (2009) Jonathan D Finn et al. MOLECULAR THERAPY
- Adeno-Associated Virus (AAV) Serotype 9 Provides Global Cardiac Gene Transfer Superior to AAV1, AAV6, AAV7, and AAV8 in the Mouse and Rat
- (2008) Lawrence T. Bish et al. HUMAN GENE THERAPY
- Disruption of the CFTR Gene Produces a Model of Cystic Fibrosis in Newborn Pigs
- (2008) C. S. Rogers et al. SCIENCE
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