Xaysongkhame Phongsavanh

Neonatal Delivery of Exon Skipping Therapeutics Maximizes Brain Dystrophin Rescue in the Mdx52 Mouse Model of Duchenne Muscular Dystrophy

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.

Improving angiogenesis ameliorates the efficacy of ASO-based exon-skipping for the treatment of Duchenne muscular dystrophy

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

Ineffective behavioral rescue despite partial brain Dp427 restoration by AAV9-U7-mediated exon 51 skipping in mdx52 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.