Mathilde Doisy

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.

This study uses spatial transcriptomics to evaluate exon-skipping therapies for DMD in the CNS, showing U7ex51 effectively restores full-length dystrophin isoforms in neurons.

This study shows that improving angiogenesis enhances ASO-based exon-skipping therapy in DMD by increasing ASO delivery and dystrophin expression in mdx mice.

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.