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dc.creatorGuo, Ping
dc.creatorZhang, Junping
dc.creatorChrzanowski, Matthew
dc.creatorHuang, Jianhe
dc.creatorChew, Helen
dc.creatorFirrman, Jenni
dc.creatorSang, Nianli
dc.creatorDiao, Yong
dc.creatorXIAO, WEIDONG
dc.date.accessioned2023-06-22T15:11:38Z
dc.date.available2023-06-22T15:11:38Z
dc.date.issued2019-01-02
dc.identifier.issn2329-0501
dc.identifier.doihttp://dx.doi.org/10.34944/dspace/8749
dc.identifier.urihttp://hdl.handle.net/20.500.12613/8785
dc.description.abstractRecombinant adeno-associated virus (rAAV) has been developed as a successful vector for both basic research and human gene therapy. However, neutralizing antibodies (NAbs) against AAV capsids can abolish AAV infectivity on target cells, reducing the transduction efficacy. Absence of AAV NAb has become a prerequisite qualification for patients enrolled in gene therapy trials. Nevertheless, accurate assessment of AAV NAb has remained a challenging task. Here we developed a rapid assay based on the observations that AAV NAb inhibits rAAV binding to the host cell surface and NAb titers are negatively related to the amount of AAV genomes binding to the target cells. By quantifying the AAV genome on the target cells in the presence of anti-sera, AAV NAb titers can be accurately determined. The titer determined by this assay correlates well with the classical transduction-based assays. A major advantage of this method is that it can be carried out with a 30-min binding assay without the lengthy wait for a transduction outcome. This assay is independent of transduction performance of AAV serotype in the target cells. Therefore, the AAV cell-binding assay for NAb determination offers an alternative method for in vivo NAb assay.
dc.format.extent7 pages
dc.languageEnglish
dc.language.isoeng
dc.relation.ispartofFaculty/ Researcher Works
dc.relation.haspartMolecular Therapy - Methods and Clinical Development, Vol. 13
dc.relation.isreferencedbyCell Press
dc.rightsAttribution-NonCommercial-NoDerivs CC BY-NC-ND
dc.rights.urihttps://creativecommons.org/licenses/by-nc-nd/4.0/
dc.subjectAAV
dc.subjectNeutralizing antibody assay
dc.subjectGene therapy
dc.subjectVector
dc.titleRapid AAV-Neutralizing Antibody Determination with a Cell-Binding Assay
dc.typeText
dc.type.genreJournal article
dc.contributor.groupSol Sherry Thrombosis Research Center (Temple University)
dc.contributor.groupCardiovascular Research Center (Temple University)
dc.description.departmentMicrobiology and Immunology
dc.description.departmentClinical Sciences
dc.relation.doihttps://doi.org/10.1016/j.omtm.2018.11.007
dc.ada.noteFor Americans with Disabilities Act (ADA) accommodation, including help with reading this content, please contact scholarshare@temple.edu
dc.description.schoolcollegeLewis Katz School of Medicine
dc.creator.orcidZhang|0000-0001-9566-3875
dc.creator.orcidChrzanowski|0000-0001-6161-2084
dc.creator.orcidFirrman|0000-0002-9586-5204
dc.creator.orcidXiao|0000-0002-9300-980X
dc.temple.creatorGuo, Ping
dc.temple.creatorZhang, Junping
dc.temple.creatorChrzanowski, Matthew
dc.temple.creatorHuang, Jianhe
dc.temple.creatorChew, Helen
dc.temple.creatorFirrman, Jenni A.
dc.temple.creatorXiao, Weidong
refterms.dateFOA2023-06-22T15:11:38Z


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