Partial restoration of brain dystrophin by tricyclo-DNA antisense oligonucleotides alleviates emotional deficits in mdx52 mice

• Main Authors: Amel Saoudi (Author), Sacha Barberat (Author), Olivier le Coz (Author), Ophélie Vacca (Author), Mathilde Doisy Caquant (Author), Thomas Tensorer (Author), Eric Sliwinski (Author), Luis Garcia (Author), Francesco Muntoni (Author), Cyrille Vaillend (Author), Aurélie Goyenvalle (Author)
• Format: Book
• Published: Elsevier, 2023-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

The mdx52 mouse model recapitulates a frequent mutation profile associated with brain involvement in Duchenne muscular dystrophy. Deletion of exon 52 impedes expression of two dystrophins (Dp427, Dp140) expressed in brain, and is eligible for therapeutic exon-skipping strategies. We previously showe...