Dose- and genotype-dependent cardiac arrhythmia and sudden death in rats following microdystrophin gene therapy
Year: 2025
Type: article
Abstract: vg/kg, for which a causal link to GNT0004 cannot be excluded. At this dose, increased arrhythmic risk and cardiac pathological remodeling were observed. These observations highlight the potential risk of MD overexpression in the heart and suggest a need for careful monitoring of patients with DMD treated with gene therapy.
Source: Molecular Therapy
Authors: Caroline Le Guiner, Gilles Toumaniantz, Thibaut Larcher, Sylvie Marchand, Laurine Buscara, Grégory Cedrone, Cladice Varela Moreira, Amandine Lancelot, Christophe Georger, Aude Lafoux, Célia Couzinié, David Augereau, Corinne Jounier, Agnès Hivonnait, Estelle Creoff, Stéphanie Blaie, Corinne Huchet, Oumeya Adjali, Nathalie Daniele, Gérald Perret, Serge Braun
Institutions: Inserm, Centre Hospitalier Universitaire de Nantes, Gene Therapy Laboratory, Translational Research in Gene Therapy, Université de Nantes, Nantes Université, Centre National de la Recherche Scientifique, Institut du Thorax, Institut National de Recherche pour l'Agriculture, l'Alimentation et l'Environnement, Oniris, Physiopathologie Animale et bioThérapie du muscle et du système nerveux, Genethon (France), Association Francaise contre les Myopathies
Language: English
Subfield: Cardiology and Cardiovascular Medicine
Field: Medicine
Domain: Health Sciences
Open Access status: hybrid
Estimated APC paid: $4,500
Funder: Sarepta Therapeutics
OpenAlex ID: W4415517986