Scientists have evaluated a gene therapy based on micro-utrophin, a protein similar to dystrophin, which is deficient in Duchenne muscular dystrophy (DMD). This therapy was administered to mice models of the disease, exposed to pharmacological and exercise-related stress.
- The therapy provided protection against cardiac damage, as evidenced in particular by a decrease in troponin levels.
- It also improved the physical performance of mice subjected to exercise and prevented the cardiac hypertrophy and fibrosis observed in untreated mice.
- The cardioprotection was long-lasting, with benefits persisting for up to 10 months after administration.
These promising results also suggest the possibility of improving heart disease in children with DMD.
