Molecule of the Month
May 2015
Functional Correction in Mouse Models of Muscular Dystrophy Using Exon-Skipping Tricyclo-DNA Oligomers

It is shown that systemic delivery of tcDNA promotes a high degree of rescue of dystrophin expression in skeletal muscles, the heart and, to a lesser extent, the brain.
This makes tcDNA attractive as a potential future therapy for patients with DMD and other neuromuscular disorders.

This work was carried out in the group of Prof. Christian Leumann.


  • A. Goyenvalle, G. Griffith, A. Babbs, S. El Andaloussi, K. Ezzat, A. Avril, B. Dugovic, R. Chaussenot, A. Ferry, T. Voit, H. Amthor, C. Bühr, S. Schürch, M. J. A. Wood, K. E. Davies, C. Vaillend, C. Leumann, L. Garcia;
    "Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers "
    Nature Medicine, 21, 270-275, (2015); doi:10.1038/nm.3765.