Related references
Note: Only part of the references are listed.Stem cell-based therapies for Duchenne muscular dystrophy
Congshan Sun et al.
EXPERIMENTAL NEUROLOGY (2020)
Integrating gene delivery and gene-editing technologies by adenoviral vector transfer of optimized CRISPR-Cas9 components
Ignazio Maggio et al.
GENE THERAPY (2020)
Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy
Christopher E. Nelson et al.
NATURE MEDICINE (2019)
Therapeutic developments for Duchenne muscular dystrophy
Ingrid E. C. Verhaart et al.
NATURE REVIEWS NEUROLOGY (2019)
High levels of AAV vector integration into CRISPR-induced DNA breaks
Killian S. Hanlon et al.
NATURE COMMUNICATIONS (2019)
Questions Answered and Unanswered by the First CRISPR Editing Study in a Canine Model of Duchenne Muscular Dystrophy
Nalinda B. Wasala et al.
HUMAN GENE THERAPY (2019)
One-Vector System for Multiplexed CRISPR/Cas9 against Hepatitis B Virus cccDNA Utilizing High-Capacity Adenoviral Vectors
Maren Schiwon et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2018)
DNA, RNA, and Protein Tools for Editing the Genetic Information in Human Cells
Xiaoyu Chen et al.
ISCIENCE (2018)
The Impact of Chromatin Dynamics on Cas9-Mediated Genome Editing in Human Cells
Rene M. Daer et al.
ACS SYNTHETIC BIOLOGY (2017)
Genome engineering: a new approach to gene therapy for neuromuscular disorders
Christopher E. Nelson et al.
NATURE REVIEWS NEUROLOGY (2017)
CRISPR/Cas9 delivery with one single adenoviral vector devoid of all viral genes
Eric Ehrke-Schulz et al.
SCIENTIFIC REPORTS (2017)
A Systematic Review and Meta-analysis on the Epidemiology of the Muscular Dystrophies
Jean K. Mah et al.
CANADIAN JOURNAL OF NEUROLOGICAL SCIENCES (2016)
Rationally engineered Cas9 nucleases with improved specificity
Ian M. Slaymaker 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)
CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice
Li Xu et al.
MOLECULAR THERAPY (2016)
Probing the impact of chromatin conformation on genome editing tools
Xiaoyu Chen et al.
NUCLEIC ACIDS RESEARCH (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)
The emerging role of viral vectors as vehicles for DMD gene editing
Ignazio Maggio et al.
GENOME MEDICINE (2016)
Adenoviral vectors encoding CRISPR/Cas9 multiplexes rescue dystrophin synthesis in unselected populations of DMD muscle cells
Ignazio Maggio et al.
SCIENTIFIC REPORTS (2016)
The Pathogenesis and Therapy of Muscular Dystrophies
Simon Guiraud et al.
ANNUAL REVIEW OF GENOMICS AND HUMAN GENETICS, VOL 16 (2015)
The TREAT-NMD DMD Global Database: Analysis of More than 7,000 Duchenne Muscular Dystrophy Mutations
Catherine L. Bladen et al.
HUMAN MUTATION (2015)
Correction of Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients Through Genomic Excision of Exon 51 by Zinc Finger Nucleases
David G. Ousterout et al.
MOLECULAR THERAPY (2015)
Genome editing at the crossroads of delivery, specificity, and fidelity
Ignazio Maggio et al.
TRENDS IN BIOTECHNOLOGY (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)
Becker muscular dystrophy severity is linked to the structure of dystrophin
Aurelie Nicolas et al.
HUMAN MOLECULAR GENETICS (2015)
Optimizing sgRNA structure to improve CRISPR-Cas9 knockout efficiency
Ying Dang et al.
GENOME BIOLOGY (2015)
Adenoviral vector DNA for accurate genome editing with engineered nucleases
Maarten Holkers et al.
NATURE METHODS (2014)
Construction and characterization of adenoviral vectors for the delivery of TALENs into human cells
Maarten Holkers et al.
METHODS (2014)
Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA
Chengzu Long et al.
SCIENCE (2014)
The new frontier of genome engineering with CRISPR-Cas9
Jennifer A. Doudna et al.
SCIENCE (2014)
Robust ZFN-mediated genome editing in adult hemophilic mice
Xavier M. Anguela et al.
BLOOD (2013)
Development of an AdEasy-based system to produce first- and second-generation adenoviral vectors with tropism for CAR- or CD46-positive cells
Josephine M. Janssen et al.
JOURNAL OF GENE MEDICINE (2013)
Reading Frame Correction by Targeted Genome Editing Restores Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients
David G. Ousterout et al.
MOLECULAR THERAPY (2013)
Assessment of the structural and functional impact of in-frame mutations of the DMD gene, using the tools included in the eDystrophin online database
Aurelie Nicolas et al.
ORPHANET JOURNAL OF RARE DISEASES (2012)
A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity
Martin Jinek et al.
SCIENCE (2012)
In vivo genome editing restores haemostasis in a mouse model of haemophilia
Hojun Li et al.
NATURE (2011)
Immortalized pathological human myoblasts: towards a universal tool for the study of neuromuscular disorders
Kamel Mamchaoui et al.
SKELETAL MUSCLE (2011)
Serum-free transient protein production system based on adenoviral vector and PER.C6 technology: High yield and preserved bioactivity
M. J. E. Havenga et al.
BIOTECHNOLOGY AND BIOENGINEERING (2008)
Adenovirus:: from foe to friend
MAFV Gonçalves et al.
REVIEWS IN MEDICAL VIROLOGY (2006)
Transduction of myogenic cells by retargeted dual high-capacity hybrid viral vectors:: Robust dystrophin synthesis in Duchenne muscular dystrophy muscle cells
Manuel A. F. V. Goncalves et al.
MOLECULAR THERAPY (2006)
Human mesenchymal stem cells ectopically expressing full-length dystrophin can complement Duchenne muscular dystrophy myotubes by cell fusion
MAFV Gonçalves et al.
HUMAN MOLECULAR GENETICS (2006)
Endowing human adenovirus serotype 5 vectors with fiber domains of species B greatly enhances gene transfer into human mesenchymal stem cells
S Knaän-Shanzer et al.
STEM CELLS (2005)
Adeno-associated virus: from defective virus to effective vector
Manuel A. F. V. Goncalves
VIROLOGY JOURNAL (2005)
Adeno-associated virus vectors integrate at chromosome breakage sites
DG Miller et al.
NATURE GENETICS (2004)
Efficient gene targeting mediated by adeno-associated virus and DNA double-strand breaks
MH Porteus et al.
MOLECULAR AND CELLULAR BIOLOGY (2003)
CD46 is a cellular receptor for group B adenoviruses
A Gaggar et al.
NATURE MEDICINE (2003)
Chromosomal effects of adeno-associated virus vector integration
DG Miller et al.
NATURE GENETICS (2002)
Highly efficient targeted transduction of undifferentiated human hematopoietic cells by adenoviral vectors displaying fiber knobs of subgroup B
SS Knaän-Shanzer et al.
HUMAN GENE THERAPY (2001)
Efficient gene transfer into human CD34(+) cells by a retargeted adenovirus vector
DM Shayakhmetov et al.
JOURNAL OF VIROLOGY (2000)