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Severe cardiac and skeletal manifestations in DMD-edited microminipigs: an advanced surrogate for Duchenne muscular dystrophy
Duchenne muscular dystrophy (DMD) is an intractable X-linked muscular dystrophy caused by mutations in the DMD gene. While many animal models have...
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The exon junction complex is required for DMD gene splicing fidelity and myogenic differentiation
Deposition of the exon junction complex (EJC) upstream of exon-exon junctions helps maintain transcriptome integrity by preventing spurious...
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IPSC derived cardiac fibroblasts of DMD patients show compromised actin microfilaments, metabolic shift and pro-fibrotic phenotype
Duchenne muscular dystrophy (DMD) is a severe form of muscular dystrophy caused by mutations in the dystrophin gene. We characterized which isoforms...
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In Vivo Evaluation of Exon 51 Skip** in hDMD/Dmd-null Mice
Duchenne muscular dystrophy (DMD) is a fatal X-linked condition that affects 1 in 3500–6000 newborn boys a year. An out-of-frame mutation in the DMD...
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Morpholino oligonucleotide-mediated exon skip** for DMD treatment: Past insights, present challenges and future perspectives
Duchenne muscular dystrophy (DMD) is an X-linked genetic disease primarily affecting boys causing loss of the dystrophin protein, ultimately leading...
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Exons 45–55 Skip** Using Antisense Oligonucleotides in Immortalized Human DMD Muscle Cells
Antisense oligonucleotides (AOs) have demonstrated high potential as a therapy for treating genetic diseases like Duchene muscular dystrophy (DMD)....
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Altered muscle niche contributes to myogenic deficit in the D2-mdx model of severe DMD
Lack of dystrophin expression is the underlying genetic basis for Duchenne muscular dystrophy (DMD). However, disease severity varies between...
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Comprehensive analysis of genomic complexity in the 5’ end coding region of the DMD gene in patients of exons 1–2 duplications based on long-read sequencing
BackgroundDystrophinopathies are the most common X-linked inherited muscle diseases, and the disease-causing gene is DMD . Exonic duplications are a...
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Pathological alterations in the gastrointestinal tract of a porcine model of DMD
BackgroundPatients with Duchenne muscular dystrophy (DMD) develop severe skeletal and cardiac muscle pathologies, which result in premature death....
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Effect of parasitic infection on muscular function of dystrophin gene (Dmd) deficient mouse
BackgroundPrevious studies have reported many cases of Trichinella spiralis ( T. spiralis ) infection in normal skeletal muscle but there is little...
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A female carrier of a novel DMD mutation with slightly skewed X-chromosome inactivation shows a suspected case of Becker muscular dystrophy in a Chinese family
Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) are both caused by mutations in DMD gene effecting the expression of...
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Identification of two rare mutations c.1318G>A and c.6438+2T>G in a Chinese DMD family as genetic markers
BackgroundDuchenne muscular dystrophy (DMD) is a fatal X-linked recessive disorder with no effective treatment, which underscores the importance of...
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Patterned Optogenetic Stimulation Using a DMD Projector
Optical manipulation is a powerful way to control neural activity in vitro and in vivo with millisecond precision. Patterning of light provides the...
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Pannexin 1 dysregulation in Duchenne muscular dystrophy and its exacerbation of dystrophic features in mdx mice
BackgroundDuchenne muscular dystrophy (DMD) is associated with impaired muscle regeneration, progressive muscle weakness, damage, and wasting. While...
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N-terminal titin fragment: a non-invasive, pharmacodynamic biomarker for microdystrophin efficacy
BackgroundMultiple clinical trials to assess the efficacy of AAV-directed gene transfer in participants with Duchenne muscular dystrophy (DMD) are...
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Replenishing NAD+ content reduces aspects of striated muscle disease in a dog model of Duchenne muscular dystrophy
Duchenne muscular dystrophy (DMD) is an X-linked disease caused by mutations in DMD gene and loss of the protein dystrophin, which ultimately leads...
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Production of Duchenne muscular dystrophy cellular model using CRISPR-Cas9 exon deletion strategy
Duchenne Muscular Dystrophy (DMD) is a progressive muscle wasting disorder caused by loss-of-function mutations in the dystrophin gene. Although the...
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Cell Therapy Strategies on Duchenne Muscular Dystrophy: A Systematic Review of Clinical Applications
Duchenne Muscular Dystrophy (DMD) is an inherited genetic disorder characterized by progressive degeneration of muscle tissue, leading to functional...
