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

Résumé fourni par la source

mice were treated with a pro-angiogenic treatment prior to ASO administration targeting exon 23 of dystrophin pre-mRNA. Angiogenic stimulation increased capillary density and improved ASO delivery to muscles (3.8-fold), exon skipping (1.8-fold), and dystrophin expression (1.5-fold) compared to ASO alone. These molecular improvements were associated with increased myofiber size, larger mean cross-sectional area, and decreased serum myomesin levels, without signs of toxicity. This study provides proof of concept that promoting angiogenesis can enhance the potency of ASO-based treatments, offering a complementary strategy to improve therapeutic outcomes in DMD.

Ce résumé expose les affirmations des auteurs. BNTIC ne l’interprète pas comme une validation indépendante des résultats.