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dc.creatorBalciunas, D
dc.creatorWangensteen, KJ
dc.creatorWilber, A
dc.creatorBell, J
dc.creatorGeurts, A
dc.creatorSivasubbu, S
dc.creatorWang, X
dc.creatorHackett, PB
dc.creatorLargaespada, DA
dc.creatorMcIvor, RS
dc.creatorEkker, SC
dc.date.accessioned2021-02-01T22:14:04Z
dc.date.available2021-02-01T22:14:04Z
dc.date.issued2006-11-01
dc.identifier.issn1553-7390
dc.identifier.issn1553-7404
dc.identifier.doihttp://dx.doi.org/10.34944/dspace/5612
dc.identifier.other17096595 (pubmed)
dc.identifier.urihttp://hdl.handle.net/20.500.12613/5630
dc.description.abstractViruses and transposons are efficient tools for permanently delivering foreign DNA into vertebrate genomes but exhibit diminished activity when cargo exceeds 8 kilobases (kb). This size restriction limits their molecular genetic and biotechnological utility, such as numerous therapeutically relevant genes that exceed 8 kb in size. Furthermore, a greater payload capacity vector would accommodate more sophisticated cis cargo designs to modulate the expression and mutagenic risk of these molecular therapeutics. We show that the Tol2 transposon can efficiently integrate DNA sequences larger than 10 kb into human cells. We characterize minimal sequences necessary for transposition (miniTol2) in vivo in zebrafish and in vitro in human cells. Both the 8.5-kb Tol2 transposon and 5.8-kb miniTol2 engineered elements readily function to revert the deficiency of fumarylacetoacetate hydrolase in an animal model of hereditary tyrosinemia type 1. Together, Tol2 provides a novel nonviral vector for the delivery of large genetic payloads for gene therapy and other transgenic applications. © 2006 Balciunas et al.
dc.format.extent1715-1724
dc.language.isoen
dc.relation.haspartPLoS Genetics
dc.relation.isreferencedbyPublic Library of Science (PLoS)
dc.rightsCC BY
dc.subjectAnimals
dc.subjectBase Sequence
dc.subjectCells, Cultured
dc.subjectDNA Transposable Elements
dc.subjectDisease Models, Animal
dc.subjectExons
dc.subjectGene Expression
dc.subjectGene Transfer Techniques
dc.subjectHumans
dc.subjectMice
dc.subjectMolecular Sequence Data
dc.subjectTyrosinemias
dc.subjectZebrafish
dc.titleHarnessing a high cargo-capacity transposon for genetic applications in vertebrates
dc.typeArticle
dc.type.genreJournal Article
dc.relation.doi10.1371/journal.pgen.0020169
dc.ada.noteFor Americans with Disabilities Act (ADA) accommodation, including help with reading this content, please contact scholarshare@temple.edu
dc.creator.orcidBalciunas, Darius|0000-0003-1938-3243
dc.date.updated2021-02-01T22:14:01Z
refterms.dateFOA2021-02-01T22:14:04Z


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