Ontology highlight
ABSTRACT:
SUBMITTER: Majowicz A
PROVIDER: S-EPMC6586596 | biostudies-literature | 2019 Sep
REPOSITORIES: biostudies-literature
Majowicz Anna A Nijmeijer Bart B Lampen Margit H MH Spronck Lisa L de Haan Martin M Petry Harald H van Deventer Sander J SJ Meyer Christian C Tangelder Marco M Ferreira Valerie V
Molecular therapy. Methods & clinical development 20190528
Currently, individuals with pre-existing neutralizing antibodies (NABs) against adeno-associated virus (AAV) above titer of 5 are excluded from systemic AAV-based clinical trials. In this study we explored the impact of pre-existing anti-AAV5 NABs on the efficacy of AAV5-based gene therapy. AMT-060 (AAV5-human FIX) was evaluated in 10 adults with hemophilia B who tested negative for pre-existing anti-AAV5 NABs using a GFP-based assay. In this study, using a more sensitive luciferase-based assay, ...[more]