Efficient Non-Viral Integration and Stable Gene Expression in Multipotent Adult Progenitor Cells

Non-viral integrating systems, PhiC31 phage integrase (ϕC31), and Sleeping Beauty transposase (SB), provide an effective method for ex vivo gene delivery into cells. Here, we used a plasmid-encoding GFP and neomycin phosphotransferase along with recognition sequences for both ϕC31 and SB integratin...

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Main Authors: Andrew Wilber, Fernando Ulloa Montoya, Luke Hammer, Branden S. Moriarity, Aron M. Geurts, David A. Largaespada, Catherine M. Verfaillie, R. Scott McIvor, Uma Lakshmipathy
Format: Article
Language:English
Published: Wiley 2011-01-01
Series:Stem Cells International
Online Access:http://dx.doi.org/10.4061/2011/717069
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author Andrew Wilber
Fernando Ulloa Montoya
Luke Hammer
Branden S. Moriarity
Aron M. Geurts
David A. Largaespada
Catherine M. Verfaillie
R. Scott McIvor
Uma Lakshmipathy
author_facet Andrew Wilber
Fernando Ulloa Montoya
Luke Hammer
Branden S. Moriarity
Aron M. Geurts
David A. Largaespada
Catherine M. Verfaillie
R. Scott McIvor
Uma Lakshmipathy
author_sort Andrew Wilber
collection DOAJ
description Non-viral integrating systems, PhiC31 phage integrase (ϕC31), and Sleeping Beauty transposase (SB), provide an effective method for ex vivo gene delivery into cells. Here, we used a plasmid-encoding GFP and neomycin phosphotransferase along with recognition sequences for both ϕC31 and SB integrating systems to demonstrate that both systems effectively mediated integration in cultured human fibroblasts and in rat multipotent adult progenitor cells (rMAPC). Southern blot analysis of G418-resistant rMAPC clones showed a 2-fold higher number of SB-mediated insertions per clone compared to ϕC31. Sequence identification of chromosomal junction sites indicated a random profile for SB-mediated integrants and a more restricted profile for ϕC31 integrants. Transgenic rMAPC generated with both systems maintained their ability to differentiate into liver and endothelium albeit with marked attenuation of GFP expression. We conclude that both SB and ϕC31 are effective non-viral integrating systems for genetic engineering of MAPC in basic studies of stem cell biology.
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spelling doaj-art-db48085b129a49e08c0edd2a02e481002025-08-20T02:21:17ZengWileyStem Cells International1687-966X1687-96782011-01-01201110.4061/2011/717069717069Efficient Non-Viral Integration and Stable Gene Expression in Multipotent Adult Progenitor CellsAndrew Wilber0Fernando Ulloa Montoya1Luke Hammer2Branden S. Moriarity3Aron M. Geurts4David A. Largaespada5Catherine M. Verfaillie6R. Scott McIvor7Uma Lakshmipathy8Center for Genome Engineering, Gene Therapy Program, Institute of Human Genetics, University of Minnesota, Minneapolis, MN 55455, USADepartment of Medicine, Stem Cell Institute, University of Minnesota, Minneapolis, MN 55455, USADepartment of Medicine, Stem Cell Institute, University of Minnesota, Minneapolis, MN 55455, USACenter for Genome Engineering, Gene Therapy Program, Institute of Human Genetics, University of Minnesota, Minneapolis, MN 55455, USACenter for Genome Engineering, Gene Therapy Program, Institute of Human Genetics, University of Minnesota, Minneapolis, MN 55455, USACenter for Genome Engineering, Gene Therapy Program, Institute of Human Genetics, University of Minnesota, Minneapolis, MN 55455, USADepartment of Medicine, Stem Cell Institute, University of Minnesota, Minneapolis, MN 55455, USACenter for Genome Engineering, Gene Therapy Program, Institute of Human Genetics, University of Minnesota, Minneapolis, MN 55455, USADepartment of Medicine, Stem Cell Institute, University of Minnesota, Minneapolis, MN 55455, USANon-viral integrating systems, PhiC31 phage integrase (ϕC31), and Sleeping Beauty transposase (SB), provide an effective method for ex vivo gene delivery into cells. Here, we used a plasmid-encoding GFP and neomycin phosphotransferase along with recognition sequences for both ϕC31 and SB integrating systems to demonstrate that both systems effectively mediated integration in cultured human fibroblasts and in rat multipotent adult progenitor cells (rMAPC). Southern blot analysis of G418-resistant rMAPC clones showed a 2-fold higher number of SB-mediated insertions per clone compared to ϕC31. Sequence identification of chromosomal junction sites indicated a random profile for SB-mediated integrants and a more restricted profile for ϕC31 integrants. Transgenic rMAPC generated with both systems maintained their ability to differentiate into liver and endothelium albeit with marked attenuation of GFP expression. We conclude that both SB and ϕC31 are effective non-viral integrating systems for genetic engineering of MAPC in basic studies of stem cell biology.http://dx.doi.org/10.4061/2011/717069
spellingShingle Andrew Wilber
Fernando Ulloa Montoya
Luke Hammer
Branden S. Moriarity
Aron M. Geurts
David A. Largaespada
Catherine M. Verfaillie
R. Scott McIvor
Uma Lakshmipathy
Efficient Non-Viral Integration and Stable Gene Expression in Multipotent Adult Progenitor Cells
Stem Cells International
title Efficient Non-Viral Integration and Stable Gene Expression in Multipotent Adult Progenitor Cells
title_full Efficient Non-Viral Integration and Stable Gene Expression in Multipotent Adult Progenitor Cells
title_fullStr Efficient Non-Viral Integration and Stable Gene Expression in Multipotent Adult Progenitor Cells
title_full_unstemmed Efficient Non-Viral Integration and Stable Gene Expression in Multipotent Adult Progenitor Cells
title_short Efficient Non-Viral Integration and Stable Gene Expression in Multipotent Adult Progenitor Cells
title_sort efficient non viral integration and stable gene expression in multipotent adult progenitor cells
url http://dx.doi.org/10.4061/2011/717069
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