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 · 2025 · Molecular Therapy: Nucleic Acids
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Abstract
The mdx52 mouse model exhibits a common mutation profile associated with brain involvement in Duchenne muscular dystrophy (DMD), characterized by heightened anxiety, fearfulness, and impaired associative fear learning. Deletion of exon 52 disrupts the expression of two dystrophins found in the brain (Dp427 and Dp140) and is eligible for therapeutic exon-skipping strategies. We previously demonstrated that a single intracerebroventricular administration of an antisense oligonucleotide (ASO) targeting exon 51 of the Dmd gene could restore 5%–15% of Dp427 expression. This treatment reduced anxiet
Abstract by Ophélie Vacca; Amel Saoudi; Mathilde Doisy; Xaysongkhame Phongsavanh; Olivier Le Coz; Cathy Nagy; Julia Kuzniar; Cyrille Vaillend, Molecular Therapy: Nucleic Acids (2025) — licensed CC BY 4.0.
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Metadata source: DOAJ · DOI 10.1016/j.omtn.2025.102779