Timing matters: exon skipping therapy is most effective when initiated early in a mouse model of Duchenne muscular dystrophy
Sofia Stenler, Junyu Huang, Tirsa L.E. van Westering, Anna M. L. Coenen-Stass, Yahya E. Jad, Kaarel Krjutškov, Samir EL Andaloussi, Matthew J. A. Wood, Thomas C. Roberts
This study shows that exon skipping therapy is more effective when started early in adult mdx mice than in aged mice, leading to better dystrophin restoration and transcriptomic changes.
Molecular Therapy — Nucleic Acids · Jul 17, 2026Peer-reviewedNeuroscience