The distribution of dystrophin in the muscle fibre is not uniform. It is dependent on the cell nuclei, thus creating small functional units at the sarcolemmal level. Researchers from the University of Versailles Saint-Quentin-en-Yvelines, with the contribution of Genethon and the Institute of Myology, have carried out experiments in transgenic mice which have shown
- a compartmentalisation of dystrophin in its natural state in the muscle fibres, even more so at the myotendinous junctions,
- a better spatial redistribution of dystrophin following treatment of the mdx mouse with antisense oligonucleotides of the tricyclo-DNA type, compared to treatment with genome editing of the CRISPR-Cas9 type.
The authors emphasise the key role of myotendinous junctions in the restoration process
