4.8 Article

Correction of diverse muscular dystrophy mutations in human engineered heart muscle by single-site genome editing

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。
Article Cell Biology

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

Leonela Amoasii et al.

SCIENCE TRANSLATIONAL MEDICINE (2017)

Article Medicine, Research & Experimental

Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System

Annalisa Lattanzi et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2017)

Article Multidisciplinary Sciences

CRISPR-Cpf1 correction of muscular dystrophy mutations in human cardiomyocytes and mice

Yu Zhang et al.

SCIENCE ADVANCES (2017)

Article Cardiac & Cardiovascular Systems

In Vivo Genome Editing Restores Dystrophin Expression and Cardiac Function in Dystrophic Mice

Mona El Refaey et al.

CIRCULATION RESEARCH (2017)

Article Medicine, Research & Experimental

Functional correction of dystrophin actin binding domain mutations by genome editing

Viktoriia Kyrychenko et al.

JCI INSIGHT (2017)

Article Multidisciplinary Sciences

Rationally engineered Cas9 nucleases with improved specificity

Ian M. Slaymaker et al.

SCIENCE (2016)

Article Multidisciplinary Sciences

In vivo gene editing in dystrophic mouse muscle and muscle stem cells

Mohammadsharif Tabebordbar et al.

SCIENCE (2016)

Article Multidisciplinary Sciences

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

Christopher E. Nelson et al.

SCIENCE (2016)

Article Genetics & Heredity

Spell Checking Nature: Versatility of CRISPR/Cas9 for Developing Treatments for Inherited Disorders

Dania Wojtal et al.

AMERICAN JOURNAL OF HUMAN GENETICS (2016)

Article Genetics & Heredity

The importance of genetic diagnosis for Duchenne muscular dystrophy

Annemieke Aartsma-Rus et al.

JOURNAL OF MEDICAL GENETICS (2016)

Article Biotechnology & Applied Microbiology

CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice

Li Xu et al.

MOLECULAR THERAPY (2016)

Article Multidisciplinary Sciences

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

Benjamin P. Kleinstiver et al.

NATURE (2016)

Article Medicine, Research & Experimental

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)

Article Biochemistry & Molecular Biology

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

Bernd Zetsche et al.

Article Biotechnology & Applied Microbiology

Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

Yanfang Fu et al.

NATURE BIOTECHNOLOGY (2014)

Article Biochemical Research Methods

Efficient genome modification by CRISPR-Cas9 nickase with minimal off-target effects

Bin Shen et al.

NATURE METHODS (2014)

Article Biochemistry & Molecular Biology

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

F. Ann Ran et al.

Article Biotechnology & Applied Microbiology

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

Prashant Mali et al.

NATURE BIOTECHNOLOGY (2013)

Article Biotechnology & Applied Microbiology

DNA targeting specificity of RNA-guided Cas9 nucleases

Patrick D. Hsu et al.

NATURE BIOTECHNOLOGY (2013)

Review Genetics & Heredity

Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches

Rebecca J. Fairclough et al.

NATURE REVIEWS GENETICS (2013)

Article Multidisciplinary Sciences

RNA-Guided Human Genome Engineering via Cas9

Prashant Mali et al.

SCIENCE (2013)

Article Multidisciplinary Sciences

Multiplex Genome Engineering Using CRISPR/Cas Systems

Le Cong et al.

SCIENCE (2013)

Article Clinical Neurology

Evidence-Based Path to Newborn Screening for Duchenne Muscular Dystrophy

Jerry R. Mendell et al.

ANNALS OF NEUROLOGY (2012)

Article Multidisciplinary Sciences

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

Martin Jinek et al.

SCIENCE (2012)

Review Genetics & Heredity

Double-Strand Break End Resection and Repair Pathway Choice

Lorraine S. Symington et al.

ANNUAL REVIEW OF GENETICS, VOL 45 (2011)

Article Cardiac & Cardiovascular Systems

Cardiomyopathy in Duchenne muscular dystrophy: pathogenesis and therapeutics

Abdallah Fayssoil et al.

HEART FAILURE REVIEWS (2010)

Article Clinical Neurology

DMD pseudoexon mutations: Splicing efficiency, phenotype, and potential therapy

Olga L. Gurvich et al.

ANNALS OF NEUROLOGY (2008)