The teams responsible for developing a gene therapy (GT) mediated by a recombinant adenovirus-associate for Duchenne muscular dystrophy (DMD), delandistrogene moxeparvovec (a gene therapy authorised in the United States under the name Elevidys®), are reporting the results of immunological studies carried out following the occurrence of undesirable side-effects:
- two cases of severe myositis occurred in sick children included in the ENDEAVOR protocol aimed at having the muscle produce microdystrophin,
- the study of the immune response linked to T lymphocytes in these patients demonstrated that exons 8 and 9 of the microdystrophin transgene were immunogenic.
On the basis of these results, the authors recommend excluding DMD patients with an anomaly in exons 8 and 9, which would put them at risk of developing highly deleterious immune reactions.
