相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy
Leonela Amoasii et al.
SCIENCE TRANSLATIONAL MEDICINE (2017)
Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy
Niclas E. Bengtsson et al.
NATURE COMMUNICATIONS (2017)
CRISPR/Cas9-Mediated Genome Editing Corrects Dystrophin Mutation in Skeletal Muscle Stem Cells in a Mouse Model of Muscle Dystrophy
Pei Zhu et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2017)
Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System
Annalisa Lattanzi et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2017)
CRISPR-Cpf1 correction of muscular dystrophy mutations in human cardiomyocytes and mice
Yu Zhang et al.
SCIENCE ADVANCES (2017)
In Vivo Genome Editing Restores Dystrophin Expression and Cardiac Function in Dystrophic Mice
Mona El Refaey et al.
CIRCULATION RESEARCH (2017)
Functional correction of dystrophin actin binding domain mutations by genome editing
Viktoriia Kyrychenko et al.
JCI INSIGHT (2017)
Defined Engineered Human Myocardium With Advanced Maturation for Applications in Heart Failure Modeling and Repair
Malte Tiburcy et al.
CIRCULATION (2017)
Rationally engineered Cas9 nucleases with improved specificity
Ian M. Slaymaker et al.
SCIENCE (2016)
Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy
Chengzu Long et al.
SCIENCE (2016)
In vivo gene editing in dystrophic mouse muscle and muscle stem cells
Mohammadsharif Tabebordbar et al.
SCIENCE (2016)
In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy
Christopher E. Nelson et al.
SCIENCE (2016)
Spell Checking Nature: Versatility of CRISPR/Cas9 for Developing Treatments for Inherited Disorders
Dania Wojtal et al.
AMERICAN JOURNAL OF HUMAN GENETICS (2016)
A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells
Courtney S. Young et al.
CELL STEM CELL (2016)
The importance of genetic diagnosis for Duchenne muscular dystrophy
Annemieke Aartsma-Rus et al.
JOURNAL OF MEDICAL GENETICS (2016)
CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice
Li Xu et al.
MOLECULAR THERAPY (2016)
High-fidelity CRISPR-Cas9 nucleases with no detectable genome-wide off-target effects
Benjamin P. Kleinstiver et al.
NATURE (2016)
Selection-free gene repair after adenoviral vector transduction of designer nucleases: rescue of dystrophin synthesis in DMD muscle cell populations
Ignazio Maggio et al.
NUCLEIC ACIDS RESEARCH (2016)
Efficient Restoration of the Dystrophin Gene Reading Frame and Protein Structure in DMD Myoblasts Using the CinDel Method
Jean-Paul Iyombe-Engembe et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2016)
Adenoviral vectors encoding CRISPR/Cas9 multiplexes rescue dystrophin synthesis in unselected populations of DMD muscle cells
Ignazio Maggio et al.
SCIENTIFIC REPORTS (2016)
Cpf1 Is a Single RNA-Guided Endonuclease of a Class 2 CRISPR-Cas System
Bernd Zetsche et al.
CELL (2015)
Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy
David G. Ousterout et al.
NATURE COMMUNICATIONS (2015)
Precise Correction of the Dystrophin Gene in Duchenne Muscular Dystrophy Patient Induced Pluripotent Stem Cells by TALEN and CRISPR-Cas9
Hongmei Lisa Li et al.
STEM CELL REPORTS (2015)
Improving CRISPR-Cas nuclease specificity using truncated guide RNAs
Yanfang Fu et al.
NATURE BIOTECHNOLOGY (2014)
Efficient genome modification by CRISPR-Cas9 nickase with minimal off-target effects
Bin Shen et al.
NATURE METHODS (2014)
Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA
Chengzu Long et al.
SCIENCE (2014)
Double Nicking by RNA-Guided CRISPR Cas9 for Enhanced Genome Editing Specificity
F. Ann Ran et al.
CELL (2013)
Distinct Metabolic Flow Enables Large-Scale Purification of Mouse and Human Pluripotent Stem Cell-Derived Cardiomyocytes
Shugo Tohyama et al.
CELL STEM CELL (2013)
CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering
Prashant Mali et al.
NATURE BIOTECHNOLOGY (2013)
DNA targeting specificity of RNA-guided Cas9 nucleases
Patrick D. Hsu et al.
NATURE BIOTECHNOLOGY (2013)
Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches
Rebecca J. Fairclough et al.
NATURE REVIEWS GENETICS (2013)
RNA-Guided Human Genome Engineering via Cas9
Prashant Mali et al.
SCIENCE (2013)
Multiplex Genome Engineering Using CRISPR/Cas Systems
Le Cong et al.
SCIENCE (2013)
Evidence-Based Path to Newborn Screening for Duchenne Muscular Dystrophy
Jerry R. Mendell et al.
ANNALS OF NEUROLOGY (2012)
A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity
Martin Jinek et al.
SCIENCE (2012)
Double-Strand Break End Resection and Repair Pathway Choice
Lorraine S. Symington et al.
ANNUAL REVIEW OF GENETICS, VOL 45 (2011)
Cardiomyopathy in Duchenne muscular dystrophy: pathogenesis and therapeutics
Abdallah Fayssoil et al.
HEART FAILURE REVIEWS (2010)
Theoretic Applicability of Antisense-Mediated Exon Skipping for Duchenne Muscular Dystrophy Mutations
Annemieke Aartsma-Rus et al.
HUMAN MUTATION (2009)
DMD pseudoexon mutations: Splicing efficiency, phenotype, and potential therapy
Olga L. Gurvich et al.
ANNALS OF NEUROLOGY (2008)
Prevention of dystrophin-deficient cardiomyopathy in twenty-one-month-old carrier mice by mosaic dystrophin expression or complementary dystrophin/utrophin expression
Brian Bostick et al.
CIRCULATION RESEARCH (2008)