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)

2026-04-09

Abstract excerpt

<h4>Background: </h4> /Objectives: 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 effectiveness of an mRNA-based therapy targeting these exon deletions, which are frequently seen in DMD patients. <h4>Methods:</h4> The current study in...

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Literature Corpus work: accc4f03-64dc-5aee-9dcc-9aaeee6ec68a
DOI: 10.20944/preprints202511.0016.v2

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