The emerging role of viral vectors as vehicles for DMD gene editing

Origins of Programmable Nucleases for Genome Engineering

(2016) Srinivasan Chandrasegaran et al.   JOURNAL OF MOLECULAR BIOLOGY

Development of Novel Adenoviral Vectors to Overcome Challenges Observed With HAdV-5–based Constructs

(2016) Julio Alonso-Padilla 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

Confirmation of donor-derived dystrophin in a duchenne muscular dystrophy patient allotransplanted with normal myoblasts

(2016) Daniel Skuk et al.   MUSCLE & NERVE

An essential receptor for adeno-associated virus infection

(2016) S. Pillay et al.   NATURE

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 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

Intra-arterial transplantation of HLA-matched donor mesoangioblasts in Duchenne muscular dystrophy

(2015) G. Cossu et al.   EMBO Molecular Medicine

Advances in genetic therapeutic strategies for Duchenne muscular dystrophy

(2015) Simon Guiraud et al.   EXPERIMENTAL PHYSIOLOGY

Progress and prospects of gene therapy clinical trials for the muscular dystrophies

(2015) Niclas E. Bengtsson 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

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

Gene therapy returns to centre stage

(2015) Luigi Naldini   NATURE

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

Dystrophin expression in muscle stem cells regulates their polarity and asymmetric division

(2015) Nicolas A Dumont et al.   NATURE MEDICINE

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

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

Human Satellite Cell Transplantation and Regeneration from Diverse Skeletal Muscles

(2015) Xiaoti Xu et al.   Stem Cell Reports

Innate Immunity to Adenovirus

(2014) Rodinde Hendrickx et al.   HUMAN GENE THERAPY

Adenovirus-Based Vaccines for Fighting Infectious Diseases and Cancer: Progress in the Field

(2014) Dragomira Majhen et al.   HUMAN GENE THERAPY

A guide to genome engineering with programmable nucleases

(2014) Hyongbum Kim et al.   NATURE REVIEWS GENETICS

Engineering adeno-associated viruses for clinical gene therapy

(2014) Melissa A. Kotterman et al.   NATURE REVIEWS GENETICS

Skipping Multiple Exons of Dystrophin Transcripts Using Cocktail Antisense Oligonucleotides

(2014) Yusuke Echigoya et al.   Nucleic Acid Therapeutics

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

Recent progress in satellite cell/myoblast engraftment - relevance for therapy

(2013) Deborah Briggs et al.   FEBS Journal

Gene Correction of a Duchenne Muscular Dystrophy Mutation by Meganuclease-Enhanced Exon Knock-In

(2013) Linda Popplewell et al.   HUMAN GENE THERAPY

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

Microdystrophin Ameliorates Muscular Dystrophy in the Canine Model of Duchenne Muscular Dystrophy

(2013) Jin-Hong Shin et al.   MOLECULAR THERAPY

Satellite Cells and the Muscle Stem Cell Niche

(2013) Hang Yin et al.   PHYSIOLOGICAL REVIEWS

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

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

The emerging biology of muscle stem cells: Implications for cell-based therapies

(2012) C. Florian Bentzinger et al.   BIOESSAYS

Successful Regional Delivery and Long-term Expression of a Dystrophin Gene in Canine Muscular Dystrophy: A Preclinical Model for Human Therapies

(2012) Zejing Wang et al.   MOLECULAR THERAPY

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

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

(2012) M. Jinek et al.   SCIENCE

Current advances in cell therapy strategies for muscular dystrophies

(2011) Elisa Negroni et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Intramuscular cell transplantation as a potential treatment of myopathies: clinical and preclinical relevant data

(2011) Daniel Skuk et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Endonucleases: tools to correct the dystrophin gene

(2011) Joel Rousseau et al.   JOURNAL OF GENE MEDICINE

Stem Cell-Mediated Transfer of a Human Artificial Chromosome Ameliorates Muscular Dystrophy

(2011) F. S. Tedesco et al.   Science Translational Medicine

Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D

(2010) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

Long-Term Contribution of Human Bone Marrow Mesenchymal Stromal Cells to Skeletal Muscle Regeneration in Mice

(2010) Anabel S. De La Garza-Rodea et al.   CELL TRANSPLANTATION

Collaboration and competition between DNA double-strand break repair pathways

(2010) Elizabeth M. Kass et al.   FEBS LETTERS

Targeting DNA Double-Strand Breaks with TAL Effector Nucleases

(2010) M. Christian et al.   GENETICS

Widespread Muscle Expression of an AAV9 Human Mini-dystrophin Vector After Intravenous Injection in Neonatal Dystrophin-deficient Dogs

(2010) Joe N Kornegay et al.   MOLECULAR THERAPY

Dystrophin Immunity in Duchenne's Muscular Dystrophy

(2010) Jerry R. Mendell et al.   NEW ENGLAND JOURNAL OF MEDICINE

Substrate Elasticity Regulates Skeletal Muscle Stem Cell Self-Renewal in Culture

(2010) P. M. Gilbert et al.   SCIENCE

Limb-girdle muscular dystrophy type 2D gene therapy restores α-sarcoglycan and associated proteins

(2009) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

The 8th and 9th tandem spectrin-like repeats of utrophin cooperatively form a functional unit to interact with polarity-regulating kinase PAR-1b

(2009) Kazunari Yamashita et al.   BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS

AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells

(2009) F. Mingozzi et al.   BLOOD

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

(2009) M. L. Brantly et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Breaking the Code of DNA Binding Specificity of TAL-Type III Effectors

(2009) J. Boch et al.   SCIENCE

A Simple Cipher Governs DNA Recognition by TAL Effectors

(2009) M. J. Moscou et al.   SCIENCE

Disease-Specific Induced Pluripotent Stem Cells

(2008) In-Hyun Park et al.   CELL

Adenovirus Serotype 5 Hexon Mediates Liver Gene Transfer

(2008) Simon N. Waddington et al.   CELL

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