Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy
出版年份 2017 全文链接
标题
Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy
作者
关键词
-
出版物
Science Translational Medicine
Volume 9, Issue 418, Pages eaan8081
出版商
American Association for the Advancement of Science (AAAS)
发表日期
2017-12-12
DOI
10.1126/scitranslmed.aan8081
参考文献
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。- Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy
- (2017) Niclas E. Bengtsson et al. Nature Communications
- FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga
- (2017) Annemieke Aartsma-Rus et al. Nucleic Acid Therapeutics
- CRISPR-Cpf1 correction of muscular dystrophy mutations in human cardiomyocytes and mice
- (2017) Yu Zhang et al. Science Advances
- 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
- Distribution of dystrophin gene deletions in a Chinese population
- (2016) Yuanyuan Li et al. JOURNAL OF INTERNATIONAL MEDICAL RESEARCH
- Eteplirsen Approved for Duchenne Muscular Dystrophy: The FDA Faces a Difficult Choice
- (2016) Cy A Stein MOLECULAR THERAPY
- Analyzing CRISPR genome-editing experiments with CRISPResso
- (2016) Luca Pinello et al. NATURE BIOTECHNOLOGY
- Eteplirsen therapy for Duchenne muscular dystrophy: skipping to the front of the line
- (2016) James J. Dowling Nature Reviews Neurology
- Efficient Restoration of the Dystrophin Gene Reading Frame and Protein Structure in DMD Myoblasts Using the CinDel Method
- (2016) Jean-Paul Iyombe-Engembe et al. Molecular Therapy-Nucleic Acids
- The Pathogenesis and Therapy of Muscular Dystrophies
- (2015) Simon Guiraud et al. Annual Review of Genomics and Human Genetics
- Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virus
- (2015) Yongping Yue et al. HUMAN MOLECULAR GENETICS
- The TREAT-NMD DMD Global Database: Analysis of More than 7,000 Duchenne Muscular Dystrophy Mutations
- (2015) Catherine L. Bladen et al. HUMAN MUTATION
- Prevalence of Duchenne and Becker Muscular Dystrophies in the United States
- (2015) P. A. Romitti et al. PEDIATRICS
- 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
- Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy
- (2015) David G. Ousterout et al. Nature Communications
- Gene therapy for Duchenne muscular dystrophy
- (2015) Julian Ramos et al. Expert Opinion on Orphan Drugs
- An emerging consensus on cardiac regeneration
- (2014) Jop H van Berlo et al. NATURE MEDICINE
- Characterization of 65 Epitope-Specific Dystrophin Monoclonal Antibodies in Canine and Murine Models of Duchenne Muscular Dystrophy by Immunostaining and Western Blot
- (2014) Kasun Kodippili et al. PLoS One
- Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA
- (2014) C. Long et al. SCIENCE
- Adeno-associated viral vectors do not efficiently target muscle satellite cells
- (2014) Andrea LH Arnett et al. Molecular Therapy-Methods & Clinical Development
- Muscular dystrophies
- (2013) Eugenio Mercuri et al. LANCET
- Microdystrophin Ameliorates Muscular Dystrophy in the Canine Model of Duchenne Muscular Dystrophy
- (2013) Jin-Hong Shin et al. MOLECULAR THERAPY
- AAV Genome Loss From Dystrophic Mouse Muscles During AAV-U7 snRNA-mediated Exon-skipping Therapy
- (2013) Maëva Le Hir et al. MOLECULAR THERAPY
- Genome engineering using the CRISPR-Cas9 system
- (2013) F Ann Ran et al. Nature Protocols
- Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches
- (2013) Rebecca J. Fairclough et al. NATURE REVIEWS GENETICS
- 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
- A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity
- (2012) M. Jinek et al. SCIENCE
- Becker muscular dystrophy patients with deletions around exon 51; a promising outlook for exon skipping therapy in Duchenne patients
- (2010) A.T.J.M. Helderman-van den Enden et al. NEUROMUSCULAR DISORDERS
- A Duchenne Muscular Dystrophy Gene Hot Spot Mutation in Dystrophin-Deficient Cavalier King Charles Spaniels Is Amenable to Exon 51 Skipping
- (2010) Gemma L. Walmsley et al. PLoS One
- Dose-dependent restoration of dystrophin expression in cardiac muscle of dystrophic mice by systemically delivered morpholino
- (2009) B Wu et al. GENE THERAPY
- Partial Rescue of Growth Failure in Growth Hormone (GH)-Deficient Mice by a Single Injection of a Double-Stranded Adeno-Associated Viral Vector Expressing the GH Gene Driven by a Muscle-Specific Regulatory Cassette
- (2009) Marco Martari et al. HUMAN GENE THERAPY
- Recent developments in adeno‐associated virus vector technology
- (2008) Hildegard Büning et al. JOURNAL OF GENE MEDICINE
- Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection
- (2008) Carmela Zincarelli et al. MOLECULAR THERAPY
Create your own webinar
Interested in hosting your own webinar? Check the schedule and propose your idea to the Peeref Content Team.
Create NowBecome a Peeref-certified reviewer
The Peeref Institute provides free reviewer training that teaches the core competencies of the academic peer review process.
Get Started