Article

Four new Duchenne muscular dystrophy mouse models with clinically relevant exon deletions in the human <i>DMD</i> gene

2025-11-21

Abstract excerpt

Mutation specific therapeutic approaches, like exon skipping or gene-editing, hold promise for the treatment of Duchenne muscular dystrophy (DMD). Translatability of preclinical studies investigating these approaches could greatly be improved through the use of humanized mouse models, as these allow preclinical testing of human specific sequences. We developed four novel humanized DMD mouse models with either a de...

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Literature Corpus work: ffda320b-1216-5dc3-b14a-6659573c95fd
DOI: 10.1101/2025.11.21.689489

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