Article

Gene-corrected human iPSC-derived cardiomyocytes and skeletal muscles reveal partial dystrophin Dp427 preservation and cardiac Dp116 expression in Duchenne muscular dystrophy

2025-07-15

Abstract excerpt

<h4>Background</h4> Duchenne muscular dystrophy (DMD) is a severe X-linked neuromuscular disease caused by mutations in the DMD gene, leading to the absence or dysfunction of dystrophin. While cardiac and skeletal muscles are both affected, tissue-specific differences in disease manifestation and dystrophin regulation remain poorly understood. <h4>Methods</h4> To investigate these differences, we established a h...

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Literature Corpus work: e7bfc207-926a-5af6-ae8a-92dd9eb4e857
DOI: 10.1101/2025.07.11.25331067

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