Dose- and genotype-dependent cardiac arrhythmia and sudden death in rats following microdystrophin gene therapy.

doi:10.1016/j.ymthe.2025.10.041

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Article

Dose- and genotype-dependent cardiac arrhythmia and sudden death in rats following microdystrophin gene therapy.

Molecular therapy : the journal of the American Society of Gene Therapy - 7 Jan 2026

Le Guiner Caroline, Toumaniantz Gilles, Larcher Thibaut, Marchand Sylvie, Buscara Laurine, Cedrone Grégory, Moreira Cladice Varela, Lancelot Amandine, Georger Christophe, Lafoux Aude, Couzinié Célia, Augereau David, Jounier Corinne, Hivonnait Agnès, Creoff Estelle, Blaie Stéphanie, Huchet Corinne, Adjali Oumeya, Daniele Nathalie, Perret Gérald, Braun Serge

Abstract excerpt

Recombinant adeno-associated virus (rAAV) vectors encoding microdystrophin (MD) are a promising treatment for Duchenne muscular dystrophy (DMD). GNT0004, an rAAV2/8 vector expressing MD1, is currently being tested in patients with DMD. Here, we explored supra-optimal intravenous doses of GNT0004 (2.1 × 1014 and 4.2 × 1014 vg/kg, up to 14 times the therapeutic dose) in wild-type (WT) and DMDmdx rats. In all...

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PMID: 41137396
DOI: 10.1016/j.ymthe.2025.10.041
PMCID: PMC12925808

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