title: Determination of anti-adeno-associated virus vector neutralizing antibody titer with an in vitro reporter system.
abstract: Adeno-associated virus (AAV) vectors are a platform of choice for in vivo gene transfer applications. However, neutralizing antibodies (NAb) to AAV can be found in humans and some animal species as a result of exposure to the wild-type virus, and high-titer NAb develop following AAV vector administration. In some conditions, anti-AAV NAb can block transduction with AAV vectors even when present at low titers, thus requiring prescreening before vector administration. Here we describe an improved in vitro, cell-based assay for the determination of NAb titer in serum or plasma samples. The assay is easy to setup and sensitive and, depending on the purpose, can be validated to support clinical development of gene therapy products based on AAV vectors.
authors.affiliation: 1 Genethon, Evry 91000, France .
authors.forename: Amine
authors.initials: A
authors.lastname: Meliani
authors.forename: Christian
authors.initials: C
authors.lastname: Leborgne
authors.forename: Sabrina
authors.initials: S
authors.lastname: Triffault
authors.forename: Laurence
authors.initials: L
authors.lastname: Jeanson-Leh
authors.forename: Philippe
authors.initials: P
authors.lastname: Veron
authors.forename: Federico
authors.initials: F
authors.lastname: Mingozzi
mesh_terms: D057134:Antibodies, Neutralizing / Q000097:blood*; D000914:Antibodies, Viral / Q000097:blood*; D000229:Dependovirus; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D057809:HEK293 Cells; D006367:HeLa Cells; D006801:Humans; D015203:Reproducibility of Results; D012680:Sensitivity and Specificity
chemical_list: D057134:Antibodies, Neutralizing; D000914:Antibodies, Viral
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