Gene replacement therapies for Duchenne muscular dystrophy (DMD) are based on viral vectors such as AAV (adenovirus-associated virus). An American study carried out on 101 DMD patients shows that :
- in this cohort, the median age was 11 years,
- 48% of patients were still walking at the time of the study and 59% were on corticosteroids,
- AAV seropositivity was lower for AAV9 and AAVrh74 serotypes than for AAV8 and AAV2 serotypes,
- age or steroid use were not determinants,
- in contrast to ethnicity, African-Americans were more likely to be seropositive overall.
These differentiated serological profiles will help to better target gene therapies in DMD.
