Adenoviral vectors encoding CRISPR/Cas9 multiplexes rescue dystrophin synthesis in unselected populations of DMD muscle cells
出版年份 2016 全文链接
标题
Adenoviral vectors encoding CRISPR/Cas9 multiplexes rescue dystrophin synthesis in unselected populations of DMD muscle cells
作者
关键词
-
出版物
Scientific Reports
Volume 6, Issue 1, Pages -
出版商
Springer Nature
发表日期
2016-11-15
DOI
10.1038/srep37051
参考文献
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注意:仅列出部分参考文献,下载原文获取全部文献信息。- 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
- Current Progress in Therapeutic Gene Editing for Monogenic Diseases
- (2016) Versha Prakash et al. MOLECULAR THERAPY
- Engineered Viruses as Genome Editing Devices
- (2016) Xiaoyu Chen et al. MOLECULAR THERAPY
- CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice
- (2016) Li Xu et al. MOLECULAR THERAPY
- Selection-free gene repair after adenoviral vector transduction of designer nucleases: rescue of dystrophin synthesis in DMD muscle cell populations
- (2016) Ignazio Maggio et al. NUCLEIC ACIDS RESEARCH
- The emerging role of viral vectors as vehicles for DMD gene editing
- (2016) Ignazio Maggio et al. Genome Medicine
- The Pathogenesis and Therapy of Muscular Dystrophies
- (2015) Simon Guiraud et al. Annual Review of Genomics and Human Genetics
- Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy
- (2015) J. W. McGreevy et al. Disease Models & Mechanisms
- Advances in genetic therapeutic strategies for Duchenne muscular dystrophy
- (2015) Simon Guiraud et al. EXPERIMENTAL PHYSIOLOGY
- The TREAT-NMD DMD Global Database: Analysis of More than 7,000 Duchenne Muscular Dystrophy Mutations
- (2015) Catherine L. Bladen et al. HUMAN MUTATION
- Correction of Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients Through Genomic Excision of Exon 51 by Zinc Finger Nucleases
- (2015) David G Ousterout et al. MOLECULAR THERAPY
- Invited review: Stem cells and muscle diseases: advances in cell therapy strategies
- (2015) Elisa Negroni et al. NEUROPATHOLOGY AND APPLIED NEUROBIOLOGY
- 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
- Genome editing at the crossroads of delivery, specificity, and fidelity
- (2015) Ignazio Maggio et al. TRENDS IN BIOTECHNOLOGY
- Quantifying on- and off-target genome editing
- (2015) Ayal Hendel et al. TRENDS IN BIOTECHNOLOGY
- Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy
- (2015) David G. Ousterout et al. Nature Communications
- Precise Correction of the Dystrophin Gene in Duchenne Muscular Dystrophy Patient Induced Pluripotent Stem Cells by TALEN and CRISPR-Cas9
- (2015) Hongmei Lisa Li et al. Stem Cell Reports
- Adenovirus: The First Effective In Vivo Gene Delivery Vector
- (2014) Ronald G. Crystal HUMAN GENE THERAPY
- Characterization of Genomic Deletion Efficiency Mediated by Clustered Regularly Interspaced Palindromic Repeats (CRISPR)/Cas9 Nuclease System in Mammalian Cells
- (2014) Matthew C. Canver et al. JOURNAL OF BIOLOGICAL CHEMISTRY
- Construction and characterization of adenoviral vectors for the delivery of TALENs into human cells
- (2014) Maarten Holkers et al. METHODS
- Easy quantitative assessment of genome editing by sequence trace decomposition
- (2014) Eva K. Brinkman et al. NUCLEIC ACIDS RESEARCH
- Adenoviral vector delivery of RNA-guided CRISPR/Cas9 nuclease complexes induces targeted mutagenesis in a diverse array of human cells
- (2014) Ignazio Maggio et al. Scientific Reports
- Low dystrophin levels increase survival and improve muscle pathology and function in dystrophin/utrophin double-knockout mice
- (2013) Maaike van Putten et al. FASEB JOURNAL
- Recent progress in satellite cell/myoblast engraftment - relevance for therapy
- (2013) Deborah Briggs et al. FEBS Journal
- Reading Frame Correction by Targeted Genome Editing Restores Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients
- (2013) David G Ousterout et al. MOLECULAR THERAPY
- 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
- ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering
- (2013) Thomas Gaj et al. TRENDS IN BIOTECHNOLOGY
- RNA-programmed genome editing in human cells
- (2013) Martin Jinek et al. eLife
- Evidence-based path to newborn screening for duchenne muscular dystrophy
- (2012) Jerry R. Mendell et al. ANNALS OF NEUROLOGY
- Development of an AdEasy-based system to produce first- and second-generation adenoviral vectors with tropism for CAR- or CD46-positive cells
- (2012) Josephine M. Janssen et al. JOURNAL OF GENE MEDICINE
- Gene and cell-mediated therapies for muscular dystrophy
- (2012) Patryk Konieczny et al. MUSCLE & NERVE
- Differential integrity of TALE nuclease genes following adenoviral and lentiviral vector gene transfer into human cells
- (2012) Maarten Holkers et al. NUCLEIC ACIDS RESEARCH
- Assessment of the structural and functional impact of in-frame mutations of the DMD gene, using the tools included in the eDystrophin online database
- (2012) Aurélie Nicolas et al. Orphanet Journal of Rare Diseases
- A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity
- (2012) M. Jinek et al. SCIENCE
- Immortalized pathological human myoblasts: towards a universal tool for the study of neuromuscular disorders
- (2011) Kamel Mamchaoui et al. Skeletal Muscle
- Marginal Level Dystrophin Expression Improves Clinical Outcome in a Strain of Dystrophin/Utrophin Double Knockout Mice
- (2010) Dejia Li et al. PLoS One
- Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations
- (2009) Annemieke Aartsma-Rus et al. HUMAN MUTATION
- Genotype-phenotype analysis in 2,405 patients with a dystrophinopathy using the UMD-DMD database: a model of nationwide knowledgebase
- (2009) Sylvie Tuffery-Giraud et al. HUMAN MUTATION
- Follow-up of three patients with a large in-frame deletion of exons 45–55 in the Duchenne muscular dystrophy (DMD) gene
- (2008) Akinori Nakamura et al. JOURNAL OF CLINICAL NEUROSCIENCE
- Serum-free transient protein production system based on adenoviral vector and PER.C6 technology: High yield and preserved bioactivity
- (2007) M.J.E. Havenga et al. BIOTECHNOLOGY AND BIOENGINEERING
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