High-sensitivity quantification of AAV neutralization from preclinical model and human sera
Beatrix Kovács, Aron Szepesi, Viktora Szabo, Mirella Barboni, Zoltan Zsolt Nagy, Balazs Rozsa, Daniel Hillier · FENS Forum 2024
Adeno-associated virus (AAV) vectors are the principal vehicle for gene therapy. In a large share of humans, immunity against AAVs can reduce or completely inhibit transgene expression, and can lead to adverse outcomes. The level of immunity can be estimated by assaying AAV neutralization level of the blood serum. Surprisingly, there is no widely adopted sensitive assay for the quantification of AAV neutralization.Results: We developed an in vitro neutralization assay, and quantified its reproducibility and sensitivity via longitudinal evaluation of neutralization in preclinical models and hum
ePoster · Neuroscience