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dc.creatorKelich, JM
dc.creatorMa, J
dc.creatorDong, B
dc.creatorWang, Q
dc.creatorChin, M
dc.creatorMagura, CM
dc.creatorXiao, W
dc.creatorYang, W
dc.date.accessioned2021-01-29T21:51:21Z
dc.date.available2021-01-29T21:51:21Z
dc.date.issued2015-04-29
dc.identifier.issn2329-0501
dc.identifier.issn2329-0501
dc.identifier.doihttp://dx.doi.org/10.34944/dspace/5213
dc.identifier.otherV46YE (isidoc)
dc.identifier.other26665132 (pubmed)
dc.identifier.urihttp://hdl.handle.net/20.500.12613/5231
dc.description.abstract© 2015 Official journal of the American Society of Gene & Cell Therapy Adeno-associated virus (AAV) has been developed as a promising human gene therapy vector. Particularly, recombinant AAV vector (rAAV) achieves its transduction of host cells by crossing at least three physiological barriers including plasma membrane, endosomal membrane, and nuclear envelope (NE). So far, the AAV transduction mechanism has not been explored thoroughly at the single viral particle level. In this study, we employed high-speed super-resolution single-point edge-excitation sub-diffraction (SPEED) microscopy to map the events of single rAAV2 particles infecting live human cells with an unprecedented spatiotemporal resolution of 9–12 nm and 2–20 ms. Data reveal that rAAV2 particles are imported through nuclear pore complexes (NPCs) rather than nuclear membrane budding into the nucleus. Moreover, approximately 17% of the rAAV2 molecules starting from the cytoplasm successfully transverse the NPCs to reach the nucleoplasm, revealing that the NPCs act as a strict selective step for AAV delivery. This study lastly suggests a new pathway to improve AAV vectors for human gene therapy.
dc.format.extent15047-
dc.language.isoen
dc.relation.haspartMolecular Therapy - Methods and Clinical Development
dc.relation.isreferencedbyElsevier BV
dc.rightsCC BY-NC-SA
dc.rights.urihttp://creativecommons.org/licenses/by-nc-sa/4.0/
dc.subject0601 Biochemistry and Cell Biology
dc.subjectBiomedical
dc.subjectBasic Science
dc.subjectBioengineering
dc.subjectGene Therapy
dc.subjectBiotechnology
dc.subjectGenetics
dc.subjectGeneric Health Relevance
dc.subject5.2 Cellular and gene therapies
dc.titleSuper-resolution imaging of nuclear import of adeno-associated virus in live cells
dc.typeArticle
dc.type.genreJournal Article
dc.relation.doi10.1038/mtm.2015.47
dc.ada.noteFor Americans with Disabilities Act (ADA) accommodation, including help with reading this content, please contact scholarshare@temple.edu
dc.creator.orcidYang, Weidong|0000-0002-3554-3035
dc.date.updated2021-01-29T21:51:17Z
refterms.dateFOA2021-01-29T21:51:22Z


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