Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy

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

Genome-wide target specificities of CRISPR-Cas9 nucleases revealed by multiplex Digenome-seq

(2016) Daesik Kim et al.   GENOME RESEARCH

Genome Engineering Using Adeno-associated Virus: Basic and Clinical Research Applications

(2016) Thomas Gaj et al.   MOLECULAR THERAPY

CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice

(2016) Li Xu et al.   MOLECULAR THERAPY

Genome-editing Technologies for Gene and Cell Therapy

(2016) Morgan L Maeder et al.   MOLECULAR THERAPY

High-fidelity CRISPR–Cas9 nucleases with no detectable genome-wide off-target effects

(2016) Benjamin P. Kleinstiver et al.   NATURE

Creating and evaluating accurate CRISPR-Cas9 scalpels for genomic surgery

(2016) Mehmet Fatih Bolukbasi et al.   NATURE METHODS

Engineering Delivery Vehicles for Genome Editing

(2016) Christopher E. Nelson et al.   Annual Review of Chemical and Biomolecular Engineering

Engineering AAV receptor footprints for gene therapy

(2016) Victoria J Madigan et al.   Current Opinion in Virology

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

Coaxing stem cells for skeletal muscle repair

(2015) Karl J.A. McCullagh et al.   ADVANCED DRUG DELIVERY REVIEWS

Exon skipping therapy for Duchenne muscular dystrophy

(2015) Ryszard Kole et al.   ADVANCED DRUG DELIVERY REVIEWS

The Pathogenesis and Therapy of Muscular Dystrophies

(2015) Simon Guiraud et al.   Annual Review of Genomics and Human Genetics

Viral vector-mediated gene therapies

(2015) Katrin Hollinger et al.   CURRENT OPINION IN NEUROLOGY

An update on RNA-targeting therapies for neuromuscular disorders

(2015) Silvana Jirka et al.   CURRENT OPINION IN NEUROLOGY

An overview of the clinical application of antisense oligonucleotides for RNA-targeting therapies

(2015) Graham McClorey et al.   CURRENT OPINION IN PHARMACOLOGY

Cardiomyopathy in Duchenne Muscular Dystrophy: Current Value of Clinical, Electrophysiological and Imaging Findings in Children and Teenagers

(2015) S. Dittrich et al.   KLINISCHE PADIATRIE

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

Duchenne Muscular Dystrophy: From Diagnosis to Therapy

(2015) Maria Falzarano et al.   MOLECULES

Current Understanding of Molecular Pathology and Treatment of Cardiomyopathy in Duchenne Muscular Dystrophy

(2015) Tirsa van Westering et al.   MOLECULES

The science to look out for in 2016

(2015) Elizabeth Gibney   NATURE

Gene therapy returns to centre stage

(2015) Luigi Naldini   NATURE

Increasing the efficiency of homology-directed repair for CRISPR-Cas9-induced precise gene editing in mammalian cells

(2015) Van Trung Chu et al.   NATURE BIOTECHNOLOGY

Therapeutic genome editing: prospects and challenges

(2015) David Benjamin Turitz Cox et al.   NATURE MEDICINE

Rationally engineered Cas9 nucleases with improved specificity

(2015) I. M. Slaymaker et al.   SCIENCE

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

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

Synthetic Zinc Finger Proteins: The Advent of Targeted Gene Regulation and Genome Modification Technologies

(2014) Charles A. Gersbach et al.   ACCOUNTS OF CHEMICAL RESEARCH

Development and Applications of CRISPR-Cas9 for Genome Engineering

(2014) Patrick D. Hsu et al.   CELL

New developments in exon skipping and splice modulation therapies for neuromuscular diseases

(2014) Aleksander Touznik et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Full-Length Dystrophin Reconstitution with Adeno-Associated Viral Vectors

(2014) William Lostal et al.   HUMAN GENE THERAPY

Translational and Regulatory Challenges for Exon Skipping Therapies

(2014) Annemieke Aartsma-Rus et al.   HUMAN GENE THERAPY

Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study

(2014) Thomas Voit et al.   LANCET NEUROLOGY

Forelimb Treatment in a Large Cohort of Dystrophic Dogs Supports Delivery of a Recombinant AAV for Exon Skipping in Duchenne Patients

(2014) Caroline Le Guiner et al.   MOLECULAR THERAPY

GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases

(2014) Shengdar Q Tsai et al.   NATURE BIOTECHNOLOGY

Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice

(2014) Nicolas Wein et al.   NATURE MEDICINE

Six-Minute Walk Test: Reference Values and Prediction Equation in Healthy Boys Aged 5 to12 Years

(2014) Nathalie Goemans et al.   PLoS One

Long Term Natural History Data in Ambulant Boys with Duchenne Muscular Dystrophy: 36-Month Changes

(2014) Marika Pane et al.   PLoS One

Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA

(2014) C. Long et al.   SCIENCE

Eteplirsen for the treatment of Duchenne muscular dystrophy

(2013) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

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

(2013) Linda Popplewell et al.   HUMAN GENE THERAPY

Triple Trans-Splicing Adeno-Associated Virus Vectors Capable of Transferring the Coding Sequence for Full-Length Dystrophin Protein into Dystrophic Mice

(2013) Taeyoung Koo 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

THE 6-minute walk test and other endpoints in Duchenne muscular dystrophy: Longitudinal natural history observations over 48 weeks from a multicenter study

(2013) Craig M. Mcdonald et al.   MUSCLE & NERVE

Ambulatory capacity and disease progression as measured by the 6-minute-walk-distance in Duchenne muscular dystrophy subjects on daily corticosteroids

(2013) Nathalie Goemans et al.   NEUROMUSCULAR DISORDERS

ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering

(2013) Thomas Gaj et al.   TRENDS IN BIOTECHNOLOGY

An ex vivo gene therapy approach to treat muscular dystrophy using inducible pluripotent stem cells

(2013) Antonio Filareto et al.   Nature Communications

Human ES- and iPS-Derived Myogenic Progenitors Restore DYSTROPHIN and Improve Contractility upon Transplantation in Dystrophic Mice

(2012) Radbod Darabi et al.   Cell Stem Cell

Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping

(2012) Aurélie Goyenvalle et al.   HUMAN MOLECULAR GENETICS

AAV micro-dystrophin gene therapy alleviates stress-induced cardiac death but not myocardial fibrosis in >21-m-old mdx mice, an end-stage model of Duchenne muscular dystrophy cardiomyopathy

(2012) Brian Bostick et al.   JOURNAL OF MOLECULAR AND CELLULAR CARDIOLOGY

Long-term benefits and adverse effects of intermittent versus daily glucocorticoids in boys with Duchenne muscular dystrophy

(2012) V. Ricotti et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

Muscle Function Recovery in Golden Retriever Muscular Dystrophy After AAV1-U7 Exon Skipping

(2012) Adeline Vulin et al.   MOLECULAR THERAPY

TALENs: a widely applicable technology for targeted genome editing

(2012) J. Keith Joung et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

Rescue of nonsense mutations by amlexanox in human cells

(2012) Sara Gonzalez-Hilarion et al.   Orphanet Journal of Rare Diseases

Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery

(2012) Y. Aoki et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Site-specific gene correction of a point mutation in human iPS cells derived from an adult patient with sickle cell disease

(2011) J. Zou et al.   BLOOD

Meganucleases and Other Tools for Targeted Genome Engineering: Perspectives and Challenges for Gene Therapy

(2011) George Silva et al.   CURRENT GENE THERAPY

Chemical and Mechanistic Toxicology Evaluation of Exon Skipping Phosphorodiamidate Morpholino Oligomers in mdx Mice

(2011) Peter Sazani et al.   INTERNATIONAL JOURNAL OF TOXICOLOGY

Repeat-Dose Toxicology Evaluation in Cynomolgus Monkeys of AVI-4658, a Phosphorodiamidate Morpholino Oligomer (PMO) Drug for the Treatment of Duchenne Muscular Dystrophy

(2011) Peter Sazani et al.   INTERNATIONAL JOURNAL OF TOXICOLOGY

Long-term functional adeno-associated virus-microdystrophin expression in the dystrophic CXMDj dog

(2011) Taeyoung Koo et al.   JOURNAL OF GENE MEDICINE

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

(2011) Sebahattin Cirak et al.   LANCET

AAV-microdystrophin Therapy Improves Cardiac Performance in Aged Female mdx Mice

(2011) Brian Bostick et al.   MOLECULAR THERAPY

Long-term Restoration of Cardiac Dystrophin Expression in Golden Retriever Muscular Dystrophy Following rAAV6-mediated Exon Skipping

(2011) Lawrence T Bish et al.   MOLECULAR THERAPY

In vivo genome editing restores haemostasis in a mouse model of haemophilia

(2011) Hojun Li et al.   NATURE

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

(2011) Nathalie M. Goemans et al.   NEW ENGLAND JOURNAL OF MEDICINE

In Situ Genetic Correction of the Sickle Cell Anemia Mutation in Human Induced Pluripotent Stem Cells Using Engineered Zinc Finger Nucleases

(2011) Vittorio Sebastiano et al.   STEM CELLS

Diagnosis and management of Duchenne muscular dystrophy

(2010) PETER BAXTER   DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY

Meganucleases can restore the reading frame of a mutated dystrophin

(2010) P Chapdelaine et al.   GENE THERAPY

Safety Pharmacology and Genotoxicity Evaluation of AVI-4658

(2010) Peter Sazani et al.   INTERNATIONAL JOURNAL OF TOXICOLOGY

Preclinical PK and PD Studies on 2′-O-Methyl-phosphorothioate RNA Antisense Oligonucleotides in the mdx Mouse Model

(2010) Hans Heemskerk et al.   MOLECULAR THERAPY

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

The Status of Exon Skipping as a Therapeutic Approach to Duchenne Muscular Dystrophy

(2010) Qi-Long Lu et al.   MOLECULAR THERAPY

A TALE nuclease architecture for efficient genome editing

(2010) Jeffrey C Miller et al.   NATURE BIOTECHNOLOGY

Genome editing with engineered zinc finger nucleases

(2010) Fyodor D. Urnov et al.   NATURE REVIEWS GENETICS

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

Dystrophin Immunity in Duchenne's Muscular Dystrophy

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

Past, present and future of myoblast transplantation in the treatment of Duchenne muscular dystrophy

(2010) Beniamino Palmieri et al.   PEDIATRIC TRANSPLANTATION

The I-CreI meganuclease and its engineered derivatives: applications from cell modification to gene therapy

(2010) S. Arnould et al.   PROTEIN ENGINEERING DESIGN & SELECTION

Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs

(2009) Toshifumi Yokota et al.   ANNALS OF NEUROLOGY

Mutational spectrum of DMD mutations in dystrophinopathy patients: application of modern diagnostic techniques to a large cohort

(2009) Kevin M. Flanigan et al.   HUMAN MUTATION

Cell based therapy for duchenne muscular dystrophy

(2009) Andrea Farini et al.   JOURNAL OF CELLULAR PHYSIOLOGY

The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy

(2009) Craig M. McDonald et al.   MUSCLE & NERVE

Adeno-Associated Virus Serotype-9 Microdystrophin Gene Therapy Ameliorates Electrocardiographic Abnormalities in mdx Mice

(2008) Brian Bostick et al.   HUMAN GENE THERAPY

Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function

(2008) HaiFang Yin et al.   HUMAN MOLECULAR GENETICS

Systemic humanminidystrophingene transfer improves functions and life span of dystrophin and dystrophin/utrophin-deficient mice

(2008) Bing Wang et al.   JOURNAL OF ORTHOPAEDIC RESEARCH

Adeno-Associated Virus Vector Genomes Persist as Episomal Chromatin in Primate Muscle

(2008) M. Penaud-Budloo et al.   JOURNAL OF VIROLOGY

Systemic Microdystrophin Gene Delivery Improves Skeletal Muscle Structure and Function in Old Dystrophic mdx Mice

(2008) Paul Gregorevic et al.   MOLECULAR THERAPY

Transduction Efficiency and Immune Response Associated With the Administration of AAV8 Vector Into Dog Skeletal Muscle

(2008) Sachiko Ohshima et al.   MOLECULAR THERAPY

Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer

(2008) B. Wu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA