Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy
出版年份 2018 全文链接
标题
Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy
作者
关键词
-
出版物
SCIENCE
Volume -, Issue -, Pages eaau1549
出版商
American Association for the Advancement of Science (AAAS)
发表日期
2018-08-31
DOI
10.1126/science.aau1549
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。- Characterising the skeletal muscle histological phenotype of the DeltaE50-MD dog, a preclinical model of Duchenne muscular dystrophy
- (2018) J. Hildyard et al. NEUROMUSCULAR DISORDERS
- Correction of diverse muscular dystrophy mutations in human engineered heart muscle by single-site genome editing
- (2018) Chengzu Long et al. Science Advances
- Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
- (2017) Jerry R. Mendell et al. NEW ENGLAND JOURNAL OF MEDICINE
- Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy
- (2017) Leonela Amoasii et al. Science Translational Medicine
- 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
- Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy
- (2016) Jerry R. Mendell et al. ANNALS OF NEUROLOGY
- 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
- Gene Editing for Duchenne Muscular Dystrophy Using the CRISPR/Cas9 Technology: The Importance of Fine-tuning the Approach
- (2016) Jacques P Tremblay et al. MOLECULAR THERAPY
- Eteplirsen Approved for Duchenne Muscular Dystrophy: The FDA Faces a Difficult Choice
- (2016) Cy A Stein MOLECULAR THERAPY
- Eteplirsen therapy for Duchenne muscular dystrophy: skipping to the front of the line
- (2016) James J. Dowling Nature Reviews Neurology
- The Pathogenesis and Therapy of Muscular Dystrophies
- (2015) Simon Guiraud et al. Annual Review of Genomics and Human Genetics
- How much dystrophin is enough: the physiological consequences of different levels of dystrophin in themdxmouse
- (2015) Caroline Godfrey et al. HUMAN MOLECULAR 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
- 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
- Low dystrophin levels in heart can delay heart failure in mdx mice
- (2014) Maaike van Putten et al. JOURNAL OF MOLECULAR AND CELLULAR CARDIOLOGY
- Easy quantitative assessment of genome editing by sequence trace decomposition
- (2014) Eva K. Brinkman et al. NUCLEIC ACIDS RESEARCH
- 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
- Mouse Embryonic Stem Cells, but Not Somatic Cells, Predominantly Use Homologous Recombination to Repair Double-Strand DNA Breaks
- (2010) Elisia D. Tichy et al. STEM CELLS AND DEVELOPMENT
- Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations
- (2009) Annemieke Aartsma-Rus et al. HUMAN MUTATION
- Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection
- (2008) Carmela Zincarelli et al. MOLECULAR THERAPY
Publish scientific posters with Peeref
Peeref publishes scientific posters from all research disciplines. Our Diamond Open Access policy means free access to content and no publication fees for authors.
Learn MoreFind the ideal target journal for your manuscript
Explore over 38,000 international journals covering a vast array of academic fields.
Search