Article

mRNA-Based Solution for 47, 48, 51, and 52 Dystrophin Exon Deletions: DMD Patient-Donate Primer Cells (In Vitro) and Transgenic Mice Experimental Study (In Vivo)

2025-11-04

Abstract excerpt

<h4>Background: </h4> Duchenne muscular dystrophy (DMD) is a genetic disorder caused by mutations in the dystrophin gene. DMD is characterized by exon deletions in about 76% of cases, with common deletions in exons 47, 48, 51, and 52. We evaluated the ef-fectiveness of an mRNA-based therapy targeting these exon deletions, which are fre-quently seen in DMD patients. <h4>Methods:</h4> The current study involved two...

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Literature Corpus work: 232185b7-f904-5724-9dad-db4ab9c08dc3
DOI: 10.20944/preprints202511.0016.v1

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