The seroprevalence of neutralizing antibodies against the adeno-associated virus capsids in Japanese hemophiliacs.

ABSTRACT: Adeno-associated virus (AAV) vectors are promising modalities of gene therapy to address unmet medical needs. However, anti-AAV neutralizing antibodies (NAbs) hamper the vector-mediated therapeutic effect. Therefore, NAb prevalence in the target population is vital in designing clinical trials with AAV vectors. Hence, updating the seroprevalence of anti-AAV NAbs, herein we analyzed sera from 100 healthy individuals and 216 hemophiliacs in Japan. In both groups, the overall seroprevalence against various AAV serotypes was 20%-30%, and the ratio of the NAb-positive population increased with age. The seroprevalence did not differ between healthy participants and hemophiliacs and was not biased by the concomitant blood-borne viral infections. The high neutralizing activity, which strongly inhi

SUBMITTER: Kashiwakura Y 

PROVIDER: S-EPMC9661668 | biostudies-literature | 2022 Dec

REPOSITORIES: biostudies-literature