Uniform sarcolemmal dystrophin expression is required to prevent extracellular microRNA release and improve dystrophic pathology

Eteplirsen treatment for Duchenne muscular dystrophy

(2018) Jay S. Charleston et al.   NEUROLOGY

Creation of Dystrophin Expressing Chimeric Cells of Myoblast Origin as a Novel Stem Cell Based Therapy for Duchenne Muscular Dystrophy

(2018) M. Siemionow et al.   Stem Cell Reviews and Reports

Creation of Dystrophin Expressing Chimeric Cells of Myoblast Origin as a Novel Stem Cell Based Therapy for Duchenne Muscular Dystrophy

(2018) M. Siemionow et al.   Stem Cell Reviews and Reports

Prevalence of Pre-existing Antibodies to CRISPR-Associated Nuclease Cas9 in the USA Population

(2018) Vijaya L. Simhadri et al.   Molecular Therapy-Methods & Clinical Development

Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy

(2018) Leonela Amoasii et al.   SCIENCE

Comprehensive RNA-Sequencing Analysis in Serum and Muscle Reveals Novel Small RNA Signatures with Biomarker Potential for DMD

(2018) Anna M.L. Coenen-Stass et al.   Molecular Therapy-Nucleic Acids

Voluntary exercise improves muscle function and does not exacerbate muscle and heart pathology in aged Duchenne muscular dystrophy mice

(2018) Bauke Kogelman et al.   JOURNAL OF MOLECULAR AND CELLULAR CARDIOLOGY

Uniform low-level dystrophin expression in the heart partially preserved cardiac function in an aged mouse model of Duchenne cardiomyopathy

(2017) Nalinda B. Wasala et al.   JOURNAL OF MOLECULAR AND CELLULAR CARDIOLOGY

Response to “Railroading at the FDA”

(2017) Francesco Muntoni et al.   NATURE BIOTECHNOLOGY

Biomarker Potential of Extracellular miRNAs in Duchenne Muscular Dystrophy

(2017) Anna M.L. Coenen-Stass et al.   TRENDS IN MOLECULAR MEDICINE

Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy

(2017) Niclas E. Bengtsson et al.   Nature Communications

Ethical guidelines for publishing in the journal of cachexia, sarcopenia and muscle: update 2017

(2017) Stephan von Haehling et al.   Journal of Cachexia Sarcopenia and Muscle

Selective release of muscle-specific, extracellular microRNAs during myogenic differentiation

(2016) Anna M.L. Coenen-Stass et al.   HUMAN MOLECULAR GENETICS

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

(2016) Ignazio Maggio et al.   Genome Medicine

Multi-level omics analysis in a murine model of dystrophin loss and therapeutic restoration

(2015) Thomas C. Roberts et al.   HUMAN MOLECULAR GENETICS

How much dystrophin is enough: the physiological consequences of different levels of dystrophin in themdxmouse

(2015) Caroline Godfrey et al.   HUMAN MOLECULAR GENETICS

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

TNF-α-Induced microRNAs Control Dystrophin Expression in Becker Muscular Dystrophy

(2015) Alyson A. Fiorillo et al.   Cell Reports

Serum Profiling Identifies Novel Muscle miRNA and Cardiomyopathy-Related miRNA Biomarkers in Golden Retriever Muscular Dystrophy Dogs and Duchenne Muscular Dystrophy Patients

(2014) Laurence Jeanson-Leh et al.   AMERICAN JOURNAL OF PATHOLOGY

Detection and quantification of extracellular microRNAs in murine biofluids

(2014) Thomas C Roberts et al.   BIOLOGICAL PROCEDURES ONLINE

Low dystrophin levels in heart can delay heart failure in mdx mice

(2014) Maaike van Putten et al.   JOURNAL OF MOLECULAR AND CELLULAR CARDIOLOGY

Biomarkers and surrogate endpoints in Duchenne: Meeting report

(2014) Annemieke Aartsma-Rus et al.   NEUROMUSCULAR DISORDERS

Assessment of RT-qPCR Normalization Strategies for Accurate Quantification of Extracellular microRNAs in Murine Serum

(2014) Thomas C. Roberts et al.   PLoS One

Circulating Muscle-specific miRNAs in Duchenne Muscular Dystrophy Patients

(2014) Xihua Li et al.   Molecular Therapy-Nucleic Acids

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

Dystrophin levels and clinical severity in Becker muscular dystrophy patients

(2013) J. C. van den Bergen et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

Extracellular microRNAs are dynamic non-vesicular biomarkers of muscle turnover

(2013) Thomas C. Roberts et al.   NUCLEIC ACIDS RESEARCH

Distinctive Serum miRNA Profile in Mouse Models of Striated Muscular Pathologies

(2013) Nicolas Vignier et al.   PLoS One

Dystromirs as Serum Biomarkers for Monitoring the Disease Severity in Duchenne Muscular Dystrophy

(2013) Irina T. Zaharieva et al.   PLoS One

Genetic characterization in symptomatic female DMD carriers: lack of relationship between X-inactivation, transcriptional DMD allele balancing and phenotype

(2012) Simona Brioschi et al.   BMC Medical Genetics

Splice Modulating Therapies for Human Disease

(2012) Pietro Spitali et al.   CELL

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

(2012) Aurélie Goyenvalle et al.   HUMAN MOLECULAR GENETICS

Loss of miR-29 in Myoblasts Contributes to Dystrophic Muscle Pathogenesis

(2012) Lijun Wang et al.   MOLECULAR THERAPY

The Effects of Low Levels of Dystrophin on Mouse Muscle Function and Pathology

(2012) Maaike van Putten et al.   PLoS One

Expression Analysis in Multiple Muscle Groups and Serum Reveals Complexity in the MicroRNA Transcriptome of the mdx Mouse with Implications for Therapy

(2012) Thomas C Roberts et al.   Molecular Therapy-Nucleic Acids

Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment

(2012) Corinne Betts et al.   Molecular Therapy-Nucleic Acids

Dystrophin quantification and clinical correlations in Becker muscular dystrophy: implications for clinical trials

(2011) K. Anthony et al.   BRAIN

miRNAs as serum biomarkers for Duchenne muscular dystrophy

(2011) Davide Cacchiarelli et al.   EMBO Molecular Medicine

Identification of Muscle-Specific MicroRNAs in Serum of Muscular Dystrophy Animal Models: Promising Novel Blood-Based Markers for Muscular Dystrophy

(2011) Hideya Mizuno et al.   PLoS One

Change in Natural History of Duchenne Muscular Dystrophy With Long-term Corticosteroid Treatment: Implications for Management

(2010) Richard T. Moxley et al.   JOURNAL OF CHILD NEUROLOGY

Clinical and genetic characterization of manifesting carriers of DMD mutations

(2010) Payam Soltanzadeh et al.   NEUROMUSCULAR DISORDERS

Xist gene regulation at the onset of X inactivation

(2009) Claire E Senner et al.   CURRENT OPINION IN GENETICS & DEVELOPMENT

Circulating microRNAs as stable blood-based markers for cancer detection

(2008) P. S. Mitchell et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA