Ineffective behavioral rescue despite partial brain Dp427 restoration by AAV9-U7-mediated exon 51 skipping in mdx52 mice
Ophélie Vacca, Amel Saoudi, Mathilde Doisy, Xaysongkhame Phongsavanh, Olivier Le Coz, Cathy Nagy, Julia Kuzniar, Cyrille Vaillend, Aurélie Goyenvalle
This study evaluates AAV9-U7-mediated exon 51 skipping in mdx52 mice to restore Dp427 and improve behavioral outcomes, finding limited Dp427 restoration and no behavioral improvement.
Molecular Therapy — Nucleic Acids · Nov 20, 2025Peer-reviewedNeuroscience