piggyBac as a high-capacity transgenesis and gene-therapy vector in human cells and mice

The stable genomic integration and expression of a large transgene is a major hurdle in gene therapy. We show that the modified piggyBac (PB) transposon system can be used to introduce a 207 kb genomic DNA fragment containing the RORγ/γt locus into human cells and mice. PB-mediated transgenesis resu...

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Detalles Bibliográficos
Autores principales: Li, Rongbo, Zhuang, Yuan, Han, Min, Xu, Tian, Wu, Xiaohui
Formato: Online Artículo Texto
Lenguaje:English
Publicado: The Company of Biologists Limited 2013
Materias:
Research Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3634665/
https://www.ncbi.nlm.nih.gov/pubmed/23519027
http://dx.doi.org/10.1242/dmm.010827
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author Li, Rongbo
Zhuang, Yuan
Han, Min
Xu, Tian
Wu, Xiaohui
author_facet Li, Rongbo
Zhuang, Yuan
Han, Min
Xu, Tian
Wu, Xiaohui
author_sort Li, Rongbo
collection PubMed
description The stable genomic integration and expression of a large transgene is a major hurdle in gene therapy. We show that the modified piggyBac (PB) transposon system can be used to introduce a 207 kb genomic DNA fragment containing the RORγ/γt locus into human cells and mice. PB-mediated transgenesis results in a single copy of a stably inherited and expressed transgene. These results indicate that PB could serve as an effective high-capacity vector for functional analysis of the mammalian genome and for gene therapy in human cells.
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spelling pubmed-36346652013-06-19 piggyBac as a high-capacity transgenesis and gene-therapy vector in human cells and mice Li, Rongbo Zhuang, Yuan Han, Min Xu, Tian Wu, Xiaohui Dis Model Mech Research Report The stable genomic integration and expression of a large transgene is a major hurdle in gene therapy. We show that the modified piggyBac (PB) transposon system can be used to introduce a 207 kb genomic DNA fragment containing the RORγ/γt locus into human cells and mice. PB-mediated transgenesis results in a single copy of a stably inherited and expressed transgene. These results indicate that PB could serve as an effective high-capacity vector for functional analysis of the mammalian genome and for gene therapy in human cells. The Company of Biologists Limited 2013-05 2013-03-08 /pmc/articles/PMC3634665/ /pubmed/23519027 http://dx.doi.org/10.1242/dmm.010827 Text en © 2013. Published by The Company of Biologists Ltd This is an Open Access article distributed under the terms of the Creative Commons Attribution Non-Commercial Share Alike License (http://creativecommons.org/licenses/by-nc-sa/3.0), which permits unrestricted non-commercial use, distribution and reproduction in any medium provided that the original work is properly cited and all further distributions of the work or adaptation are subject to the same Creative Commons License terms.
spellingShingle Research Report
Li, Rongbo
Zhuang, Yuan
Han, Min
Xu, Tian
Wu, Xiaohui
piggyBac as a high-capacity transgenesis and gene-therapy vector in human cells and mice
title piggyBac as a high-capacity transgenesis and gene-therapy vector in human cells and mice
title_full piggyBac as a high-capacity transgenesis and gene-therapy vector in human cells and mice
title_fullStr piggyBac as a high-capacity transgenesis and gene-therapy vector in human cells and mice
title_full_unstemmed piggyBac as a high-capacity transgenesis and gene-therapy vector in human cells and mice
title_short piggyBac as a high-capacity transgenesis and gene-therapy vector in human cells and mice
title_sort piggybac as a high-capacity transgenesis and gene-therapy vector in human cells and mice
topic Research Report
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3634665/
https://www.ncbi.nlm.nih.gov/pubmed/23519027
http://dx.doi.org/10.1242/dmm.010827
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