A team from the Institute of Myology has identified molecules capable of targeting NO synthase, an enzyme involved in the synthesis of nitric acid and whose expression is reduced in Becker muscular dystrophy (BMD).
The “Therapeutic Strategies Based on RNA Repair & Pathophysiology of Skeletal Muscle” group, led by France Pietri Rouxel (Myology Centre for Research) had previously demonstrated a decrease in the expression of NO synthase (NOS) in muscle biopsies of BMD patients with truncated dystrophin (BMDd45-45, deletion of exons 45-55). This is can be explained by the fact that the truncated form of dystrophin is consequently devoid of the region that usually interacts with NOS.
The team then sought to identify, in BMDd45-45 muscle biopsies, microRNAs capable of targeting NOS. Among more than 600 microRNAs analysed, two were found to be involved in the regulation of NOS: miR-708-5p and miR-34c-5p. By inhibiting these two microRNAs in myoblasts, the authors succeeded in increasing the expression of NOS.
