Application of In Vitro Cultured Primary Hepatocytes To Evaluate Species Translatability and AAV Transduction Mechanisms of Action

Scientists assessed whether in vitro recombinant adeno-associated virus (AAV) transduction assays in cultured primary hepatocytes from mice, nonhuman primates (NHP), and humans could model in vivo liver-directed AAV expression of valoctocogene roxaparvovec.