NeurosciencebioRxivPreprint
Neonatal Delivery of Exon Skipping Therapeutics Maximizes Brain Dystrophin Rescue in the Mdx52 Mouse Model of Duchenne Muscular Dystrophy
Ophélie Vacca, Mathilde Doisy, Cathy Nagy…·Aurélie Goyenvalle
The paper shows that neonatal intracerebroventricular delivery of scAAV9-U7-Ex51 provides sustained exon skipping and dystrophin restoration in the brain of mdx52 mice, outperforming systemic or intracerebroventricular ASO delivery.
DOI 10.64898/2026.10.09.757591