title: Cell-Based Measurement of Neutralizing Antibodies Against Adeno-Associated Virus (AAV).
abstract: In recent years gene therapy using adeno-associated viral (AAV) vectors to treat cardiac disease has seen an unprecedented surge, owing to its safety, low immunogenicity relative to other vectors and high and long-term transduction efficiency. This field has also been hampered by the presence of preexisting neutralizing antibodies, not only in patients participating in clinical trials but also in preclinical large animal models. These conflicting circumstances have generated the need for a simple, efficient, and fast assay to screen subjects for the presence of neutralizing antibodies, or lack thereof, in order for them to be included in gene therapy trials.
authors.affiliation: Department of Internal Medicine III, Heidelberg University Hospital, University of Heidelberg, Im Neuenheimer Feld410, 69120, Heidelberg, Germany.
authors.forename: Andreas
authors.initials: A
authors.lastname: Jungmann
authors.affiliation: Department of Internal Medicine III, Heidelberg University Hospital, University of Heidelberg, Im Neuenheimer Feld410, 69120, Heidelberg, Germany.
authors.forename: Oliver
authors.initials: O
authors.lastname: Müller
authors.affiliation: Department of Internal Medicine III, Heidelberg University Hospital, University of Heidelberg, Im Neuenheimer Feld410, 69120, Heidelberg, Germany. kleopatra.rapti@med.uni-heidelberg.de.
authors.forename: Kleopatra
authors.initials: K
authors.lastname: Rapti
mesh_terms: D000818:Animals; D057134:Antibodies, Neutralizing / Q000378:metabolism*; D002452:Cell Count; D000229:Dependovirus / Q000235:genetics / Q000276:immunology*; D016679:Genome, Viral; D006801:Humans; D009500:Neutralization Tests / Q000379:methods*; D013223:Statistics as Topic; D014161:Transduction, Genetic
chemical_list: D057134:Antibodies, Neutralizing
keywords: AAV vectors; Assay; Human and animal serum; In vitro; Neutralizing antibody