Duchenne muscular dystrophy: an updated review of common available therapies

The CRISPR Growth Spurt: from Bench to Clinic on Versatile Small RNAs

(2017) Hadi Bayat et al.   JOURNAL OF MICROBIOLOGY AND BIOTECHNOLOGY

CRISPR-Cpf1 correction of muscular dystrophy mutations in human cardiomyocytes and mice

(2017) Yu Zhang et al.   Science Advances

Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy

(2016) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

Duchenne muscular dystrophy: CRISPR/Cas9 treatment

(2016) Jerry R Mendell et al.   CELL RESEARCH

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

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

(2016) Li Xu et al.   MOLECULAR THERAPY

Histological effects of givinostat in boys with Duchenne muscular dystrophy

(2016) Paolo Bettica et al.   NEUROMUSCULAR DISORDERS

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

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

Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study

(2016) Nathalie M. Goemans et al.   PLoS One

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

Jagged 1 Rescues the Duchenne Muscular Dystrophy Phenotype

(2015) Natassia M. Vieira et al.   CELL

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

Functional disruption of the dystrophin gene in rhesus monkey using CRISPR/Cas9

(2015) Yongchang Chen et al.   HUMAN MOLECULAR GENETICS

Second-generation compound for the modulation of utrophin in the therapy of DMD

(2015) Simon Guiraud et al.   HUMAN MOLECULAR GENETICS

Taurine deficiency, synthesis and transport in the mdx mouse model for Duchenne Muscular Dystrophy

(2015) Jessica R. Terrill et al.   INTERNATIONAL JOURNAL OF BIOCHEMISTRY & CELL BIOLOGY

Model Organisms in the Fight against Muscular Dystrophy: Lessons from Drosophila and Zebrafish

(2015) Emilie Plantié et al.   MOLECULES

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

(2015) Tirsa van Westering et al.   MOLECULES

Therapeutic genome editing: prospects and challenges

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

Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers

(2015) Aurélie Goyenvalle et al.   NATURE MEDICINE

New exon-skipping strategy rescues dystrophin

(2015) Sarah Crunkhorn   NATURE REVIEWS DRUG DISCOVERY

European Medicines Agency review of ataluren for the treatment of ambulant patients aged 5 years and older with Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene

(2015) Manuel Haas et al.   NEUROMUSCULAR DISORDERS

piggyBactransposons expressing full-length human dystrophin enable genetic correction of dystrophic mesoangioblasts

(2015) Mariana Loperfido et al.   NUCLEIC ACIDS RESEARCH

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

A molecular patch for DMD

(2015) Anshu Bhardwaj   Science Translational Medicine

Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy

(2015) David G. Ousterout et al.   Nature Communications

HDAC inhibitors for muscular dystrophies: progress and prospects

(2015) M. Sandoná 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

Duchenne muscular dystrophy: current cell therapies

(2015) Dorota Sienkiewicz et al.   Therapeutic Advances in Neurological Disorders

Utrophin A is essential in mediating the functional adaptations of mdx mouse muscle following chronic AMPK activation

(2014) H. Al-Rewashdy et al.   HUMAN MOLECULAR GENETICS

Cancer epigenetics drug discovery and development: the challenge of hitting the mark

(2014) Robert M. Campbell et al.   JOURNAL OF CLINICAL INVESTIGATION

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

Histone core modifications regulating nucleosome structure and dynamics

(2014) Peter Tessarz et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

Characterization of Dystrophin Deficient Rats: A New Model for Duchenne Muscular Dystrophy

(2014) Thibaut Larcher et al.   PLoS One

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

(2014) C. Long et al.   SCIENCE

Histone deacetylase inhibitors: a potential epigenetic treatment for Duchenne muscular dystrophy

(2014) Silvia Consalvi et al.   Epigenomics

What Can We Learn From Clinical Trials of Exon Skipping for DMD?

(2014) Qi-long Lu et al.   Molecular Therapy-Nucleic Acids

Adeno-associated viral vectors do not efficiently target muscle satellite cells

(2014) Andrea LH Arnett et al.   Molecular Therapy-Methods & Clinical Development

Newborn bloodspot screening for Duchenne Muscular Dystrophy: 21 years experience in Wales (UK)

(2013) Stuart J Moat et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

New developments in the use of gene therapy to treat Duchenne muscular dystrophy

(2013) Susan Jarmin et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Dystrophin insufficiency causes selective muscle histopathology and loss of dystrophin-glycoprotein complex assembly in pig skeletal muscle

(2013) Katrin Hollinger et al.   FASEB JOURNAL

Epigenetic control of skeletal muscle regeneration

(2013) Lorenzo Giordani et al.   FEBS Journal

Highly efficient in vivo delivery of PMO into regenerating myotubes and rescue in laminin-α2 chain-null congenital muscular dystrophy mice

(2013) Yoshitsugu Aoki et al.   HUMAN MOLECULAR GENETICS

Dual AAV therapy ameliorates exercise-induced muscle injury and functional ischemia in murine models of Duchenne muscular dystrophy

(2013) Yadong Zhang et al.   HUMAN MOLECULAR GENETICS

Muscular dystrophies

(2013) Eugenio Mercuri et al.   LANCET

Unknown

(2013) Silvia Consalvi et al.   MOLECULAR MEDICINE

DNA targeting specificity of RNA-guided Cas9 nucleases

(2013) Patrick D Hsu et al.   NATURE BIOTECHNOLOGY

Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches

(2013) Rebecca J. Fairclough et al.   NATURE REVIEWS GENETICS

DNA methylation dynamics in health and disease

(2013) Yehudit Bergman et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

Chromatin Modifications as Determinants of Muscle Stem Cell Quiescence and Chronological Aging

(2013) Ling Liu et al.   Cell Reports

A Lack of Premature Termination Codon Read-Through Efficacy of PTC124 (Ataluren) in a Diverse Array of Reporter Assays

(2013) Stuart P. McElroy et al.   PLOS BIOLOGY

Nitric oxide signalling pathway in Duchenne muscular dystrophy mice: up-regulation of L-arginine transporters

(2012) Jayalakshmi Ramachandran et al.   BIOCHEMICAL JOURNAL

Myostatin: more than just a regulator of muscle mass

(2012) Josep M. Argilés et al.   DRUG DISCOVERY TODAY

An urgent need for a change in policy revealed by a study on prenatal testing for Duchenne muscular dystrophy

(2012) Apollonia T J M Helderman-van den Enden et al.   EUROPEAN JOURNAL OF HUMAN GENETICS

Perspectives of stem cell therapy in Duchenne muscular dystrophy

(2012) Mirella Meregalli et al.   FEBS Journal

Genetic Therapeutic Approaches for Duchenne Muscular Dystrophy

(2012) Helen Foster et al.   HUMAN GENE THERAPY

Nitric Oxide in Myogenesis and Therapeutic Muscle Repair

(2012) Clara De Palma et al.   MOLECULAR NEUROBIOLOGY

Sustaining Cardiac Claudin-5 Levels Prevents Functional Hallmarks of Cardiomyopathy in a Muscular Dystrophy Mouse Model

(2012) Dawn A Delfín et al.   MOLECULAR THERAPY

Engineering Multiple U7snRNA Constructs to Induce Single and Multiexon-skipping for Duchenne Muscular Dystrophy

(2012) Aurélie Goyenvalle et al.   MOLECULAR THERAPY

Epigenetic protein families: a new frontier for drug discovery

(2012) Cheryl H. Arrowsmith et al.   NATURE REVIEWS DRUG DISCOVERY

Histone methylation: a dynamic mark in health, disease and inheritance

(2012) Eric L. Greer et al.   NATURE REVIEWS GENETICS

Histone acetyltransferases and deacetylases: molecular and clinical implications to gastrointestinal carcinogenesis: Figure 1

(2011) Wei-Jian Sun et al.   ACTA BIOCHIMICA ET BIOPHYSICA SINICA

Novel Mini–Dystrophin Gene Dual Adeno-Associated Virus Vectors Restore Neuronal Nitric Oxide Synthase Expression at the Sarcolemma

(2011) Yadong Zhang et al.   HUMAN GENE THERAPY

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

Long-Term Systemic Administration of Unconjugated Morpholino Oligomers for Therapeutic Expression of Dystrophin by Exon Skipping in Skeletal Muscle: Implications for Cardiac Muscle Integrity

(2011) Alberto Malerba et al.   Nucleic Acid Therapeutics

Molecular treatments in Duchenne muscular dystrophy

(2010) Michela Guglieri et al.   CURRENT OPINION IN PHARMACOLOGY

Duchenne muscular dystrophy – What causes the increased membrane permeability in skeletal muscle?

(2010) David G. Allen et al.   INTERNATIONAL JOURNAL OF BIOCHEMISTRY & CELL BIOLOGY

Functional Rescue of Dystrophin-deficient mdx Mice by a Chimeric Peptide-PMO

(2010) HaiFang Yin et al.   MOLECULAR THERAPY

Biglycan recruits utrophin to the sarcolemma and counters dystrophic pathology in mdx mice

(2010) A. R. Amenta et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Aminoglycoside-induced mutation suppression (stop codon readthrough) as a therapeutic strategy for Duchenne muscular dystrophy

(2010) Vinod Malik et al.   Therapeutic Advances in Neurological Disorders

Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs

(2009) Toshifumi Yokota et al.   ANNALS OF NEUROLOGY

Regenerative pharmacology in the treatment of genetic diseases: The paradigm of muscular dystrophy

(2008) Chiara Mozzetta et al.   INTERNATIONAL JOURNAL OF BIOCHEMISTRY & CELL BIOLOGY