Search Results - "Wang, Derek W."
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Dantrolene enhances antisense-mediated exon skipping in human and mouse models of Duchenne muscular dystrophy
Published in Science translational medicine (12-12-2012)“…Duchenne muscular dystrophy (DMD) causes profound and progressive muscle weakness and loss, resulting in early death. DMD is usually caused by frameshifting…”
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Single nuclei transcriptomics of muscle reveals intra-muscular cell dynamics linked to dystrophin loss and rescue
Published in Communications biology (19-09-2022)“…In Duchenne muscular dystrophy, dystrophin loss leads to chronic muscle damage, dysregulation of repair, fibro-fatty replacement, and weakness. We develop…”
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Journal Article -
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Repurposing Dantrolene for Long-Term Combination Therapy to Potentiate Antisense-Mediated DMD Exon Skipping in the mdx Mouse
Published in Molecular therapy. Nucleic acids (01-06-2018)“…Duchenne muscular dystrophy (DMD) is caused by mutations in DMD, resulting in loss of dystrophin, which is essential to muscle health. DMD “exon skipping” uses…”
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