Prevalence and long-term monitoring of humoral immunity against adeno-associated virus in Duchenne Muscular Dystrophy patients

C Leborgne, V Latournerie, S Boutin, D Desgue… - Cellular …, 2019 - Elsevier
Adeno-associated virus (AAV) vectors are promising candidates for gene therapy and have
been explored as gene delivery vehicles in the treatment of Duchenne Muscular Dystrophy
(DMD). Recent studies showed compelling evidence of therapeutic efficacy in large animal
models following the intravenous delivery of AAV vectors expressing truncated forms of
dystrophin. However, to translate these results to humans, careful assessment of the
prevalence of anti-AAV neutralizing antibodies (NAbs) is needed, as presence of preexisting …