Peer-Reviewed Publication
Mol Ther Nucleic Acids2026;37(3):103017.September 8, 2026Journal Article
Timing matters: Exon skipping therapy is most effective when initiated early in a mouse model of Duchenne muscular dystrophy.
Sofia Stenler1,2, Junyu Huang3,4, Tirsa L E van Westering1,5, Anna M L Coenen-Stass1,6, Yahya Jad3,4, Kaarel Krjutškov7,8, Samir El Andaloussi9, Matthew J A Wood1,3,4,10, Thomas C Roberts1,3,4,10
1Department of Physiology, Anatomy and Genetics, University of Oxford, South Parks Road, Oxford OX1 3QX, UK.
2Uppsala University, Office for Science and Technology, 751 05 Uppsala, Sweden.
3Department of Paediatrics, University of Oxford, South Parks Road, Oxford OX1 3QX, UK.
4Institute of Developmental and Regenerative Medicine, University of Oxford, IMS-Tetsuya Nakamura Building, Old Road Campus, Roosevelt Dr, Headington, Oxford OX3 7TY, UK.
5Charles River Laboratories, Darwinweg 24, Leiden, 2333 CR South Holland, the Netherlands.
6Merck KGaA, Frankfurter Str. 250, 64293 Darmstadt, Germany.
7Celvia CC AS, 50411 Tartu, Estonia.
8Department of Obstetrics and Gynaecology, Institute of Clinical Medicine, University of Tartu, 50411 Tartu, Estonia.
9Department of Laboratory Medicine, Karolinska Institutet, 141 86 Huddinge, Sweden.
10MDUK Oxford Neuromuscular Centre, South Parks Road, Oxford, UK.
Abstract
Exon skipping is a leading therapeutic approach for Duchenne muscular dystrophy (DMD), whereby modulation of pre-mRNA splicing is used to restore the dystrophin translation reading frame. Four exon skipping drugs have received FDA accelerated approval, despite limited clinical efficacy. To investigate how treatment timing influences exon skipping outcomes, dystrophin-deficient mdx mice were inject…
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