What is the level of dystrophin expression required for effective therapy of Duchenne muscular dystrophy?

Identification of qPCR reference genes suitable for normalizing gene expression in the mdx mouse model of Duchenne muscular dystrophy

(2019) John C. W. Hildyard et al.   PLoS One

Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy

(2019) Christopher E. Nelson et al.   NATURE MEDICINE

Development of Novel Micro-dystrophins with Enhanced Functionality

(2019) Julian N. Ramos et al.   MOLECULAR THERAPY

Natural disease history of the D2-mdx mouse model for Duchenne muscular dystrophy

(2019) Maaike van Putten et al.   FASEB JOURNAL

Long-range genomic regulators of THBS1 and LTBP4 modify disease severity in Duchenne muscular dystrophy

(2018) Robert B. Weiss et al.   ANNALS OF NEUROLOGY

Tracking progress: an update on animal models for Duchenne muscular dystrophy

(2018) Dominic J. Wells   Disease Models & Mechanisms

A novel rabbit model of Duchenne muscular dystrophy generated by CRISPR/Cas9

(2018) Tingting Sui et al.   Disease Models & Mechanisms

Dystrophin's central domain forms a complex filament that becomes disorganized by in-frame deletions

(2018) Olivier Delalande et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Systemic AAV micro-dystrophin gene therapy for Duchenne muscular dystrophy

(2018) Dongsheng Duan   MOLECULAR THERAPY

Low dystrophin levels are insufficient to normalize the neuromuscular synaptic abnormalities of mdx mice

(2018) Elizabeth M. van der Pijl et al.   NEUROMUSCULAR DISORDERS

A novel high-throughput immunofluorescence analysis method for quantifying dystrophin intensity in entire transverse sections of Duchenne muscular dystrophy muscle biopsy samples

(2018) Valentina Sardone et al.   PLoS One

Use of capillary Western immunoassay (Wes) for quantification of dystrophin levels in skeletal muscle of healthy controls and individuals with Becker and Duchenne muscular dystrophy

(2018) Chantal Beekman et al.   PLoS One

A novel canine model for Duchenne muscular dystrophy (DMD): single nucleotide deletion in DMD gene exon 20

(2018) Sara Mata López et al.   Skeletal Muscle

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

(2018) Leonela Amoasii et al.   SCIENCE

Cmah-dystrophin deficient mdx mice display an accelerated cardiac phenotype that is improved following peptide-PMO exon skipping treatment

(2018) Corinne A Betts et al.   HUMAN MOLECULAR GENETICS

A rare subclinical or mild type of Becker muscular dystrophy caused by a single exon 48 deletion of the dystrophin gene

(2017) Janusz G. Zimowski et al.   JOURNAL OF APPLIED GENETICS

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

Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy

(2017) Caroline Le Guiner et al.   Nature Communications

A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy

(2017) Chady H. Hakim et al.   Molecular Therapy-Methods & Clinical Development

Association Study of Exon Variants in the NF-κB and TGFβ Pathways Identifies CD40 as a Modifier of Duchenne Muscular Dystrophy

(2016) Luca Bello et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Dystrophin Threshold Level Necessary for Normalization of Neuronal Nitric Oxide Synthase, Inducible Nitric Oxide Synthase, and Ryanodine Receptor-Calcium Release Channel Type 1 Nitrosylation in Golden Retriever Muscular Dystrophy Dystrophinopathy

(2016) Christel Gentil et al.   HUMAN GENE THERAPY

Deletion of exons 3−9 encompassing a mutational hot spot in the DMD gene presents an asymptomatic phenotype, indicating a target region for multiexon skipping therapy

(2016) Akinori Nakamura et al.   JOURNAL OF HUMAN GENETICS

Whole genome sequencing reveals a 7 base-pair deletion in DMD exon 42 in a dog with muscular dystrophy

(2016) Peter P. Nghiem et al.   MAMMALIAN GENOME

Identifying Non–Duchenne Muscular Dystrophy–Positive and False Negative Results in Prior Duchenne Muscular Dystrophy Newborn Screening Programs

(2016) Michele A. Gatheridge et al.   JAMA Neurology

Impaired regenerative capacity and lower revertant fibre expansion in dystrophin-deficient mdx muscles on DBA/2 background

(2016) Merryl Rodrigues et al.   Scientific Reports

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

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

(2015) Caroline Godfrey et al.   HUMAN MOLECULAR GENETICS

Effect of genetic background on the dystrophic phenotype inmdxmice

(2015) William D. Coley et al.   HUMAN MOLECULAR GENETICS

Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virus

(2015) Yongping Yue et al.   HUMAN MOLECULAR GENETICS

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

Elusive sources of variability of dystrophin rescue by exon skipping

(2015) Maria Candida Vila et al.   Skeletal Muscle

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

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

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

Dystrophin quantification: Biological and translational research implications

(2014) K. Anthony et al.   NEUROLOGY

A systematic review and meta-analysis on the epidemiology of Duchenne and Becker muscular dystrophy

(2014) Jean K. Mah et al.   NEUROMUSCULAR DISORDERS

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

(2014) Thibaut Larcher et al.   PLoS One

Generation of muscular dystrophy model rats with a CRISPR/Cas system

(2014) Katsuyuki Nakamura et al.   Scientific Reports

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-deficient pigs provide new insights into the hierarchy of physiological derangements of dystrophic muscle

(2013) Nikolai Klymiuk et al.   HUMAN MOLECULAR GENETICS

Evidence-based path to newborn screening for duchenne muscular dystrophy

(2012) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

A marginal level of dystrophin partially ameliorates hindlimb muscle passive mechanical properties in dystrophin-null mice

(2012) Chady H. Hakim et al.   MUSCLE & NERVE

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

(2012) Maaike van Putten et al.   PLoS One

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

(2011) K. Anthony et al.   BRAIN

Genetic Background Affects Properties of Satellite Cells and mdx Phenotypes

(2010) So-ichiro Fukada et al.   AMERICAN JOURNAL OF PATHOLOGY

Short Telomeres and Stem Cell Exhaustion Model Duchenne Muscular Dystrophy in mdx/mTR Mice

(2010) Alessandra Sacco et al.   CELL

Physiological Characterization of Muscle Strength With Variable Levels of Dystrophin Restoration in mdx Mice Following Local Antisense Therapy

(2010) Paul S Sharp et al.   MOLECULAR THERAPY

A Duchenne Muscular Dystrophy Gene Hot Spot Mutation in Dystrophin-Deficient Cavalier King Charles Spaniels Is Amenable to Exon 51 Skipping

(2010) Gemma L. Walmsley et al.   PLoS One

Marginal Level Dystrophin Expression Improves Clinical Outcome in a Strain of Dystrophin/Utrophin Double Knockout Mice

(2010) Dejia Li et al.   PLoS One

A Human-Specific Deletion in Mouse Cmah Increases Disease Severity in the mdx Model of Duchenne Muscular Dystrophy

(2010) K. Chandrasekharan et al.   Science Translational Medicine

Preservation of Muscle Force in Mdx3cv Mice Correlates with Low-Level Expression of a Near Full-Length Dystrophin Protein

(2008) Dejia Li et al.   AMERICAN JOURNAL OF PATHOLOGY

Asymptomatic Becker muscular dystrophy in a family with a multiexon deletion

(2008) Verónica Ferreiro et al.   MUSCLE & NERVE

Towards developing standard operating procedures for pre-clinical testing in the mdx mouse model of Duchenne muscular dystrophy

(2008) Miranda D. Grounds et al.   NEUROBIOLOGY OF DISEASE