Home > Publications database > An episomal DNA vector platform for the persistent genetic modification of pluripotent stem cells and their differentiated progeny. > print

001     178288
005     20240229133803.0
024 7 _ |a 10.1016/j.stemcr.2021.11.011
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041 _ _ |a English
082 _ _ |a 610
100 1 _ |a Roig-Merino, Alicia
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245 _ _ |a An episomal DNA vector platform for the persistent genetic modification of pluripotent stem cells and their differentiated progeny.
260 _ _ |a [New York, NY]
|c 2022
|b Elsevier
336 7 _ |a article
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500 _ _ |a #EA:F160#LA:F160# /2022 Jan 11;17(1):143-158
520 _ _ |a The genetic modification of stem cells (SCs) is typically achieved using integrating vectors, whose potential integrative genotoxicity and propensity for epigenetic silencing during differentiation limit their application. The genetic modification of cells should provide sustainable levels of transgene expression, without compromising the viability of a cell or its progeny. We developed nonviral, nonintegrating, and autonomously replicating minimally sized DNA nanovectors to persistently genetically modify SCs and their differentiated progeny without causing any molecular or genetic damage. These DNA vectors are capable of efficiently modifying murine and human pluripotent SCs with minimal impact and without differentiation-mediated transgene silencing or vector loss. We demonstrate that these vectors remain episomal and provide robust and sustained transgene expression during self-renewal and targeted differentiation of SCs both in vitro and in vivo through embryogenesis and differentiation into adult tissues, without damaging their phenotypic characteristics.
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650 _ 7 |a DNA vector
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650 _ 7 |a SMAR
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650 _ 7 |a differentiation
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650 _ 7 |a embryonic stem cell
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650 _ 7 |a episome
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650 _ 7 |a induced pluripotent stem cell
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650 _ 7 |a nonintegrative
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650 _ 7 |a reprogramming
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650 _ 7 |a self-renewal
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650 _ 7 |a transgenesis
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700 1 _ |a Urban, Manuela
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700 1 _ |a Bozza, Matthias
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700 1 _ |a Peterson, Julia D
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700 1 _ |a Bullen, Louise
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700 1 _ |a Büchler-Schäff, Marleen
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700 1 _ |a Stäble, Sina
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700 1 _ |a van der Hoeven, Franciscus
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700 1 _ |a Müller-Decker, Karin
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700 1 _ |a McKay, Tristan R
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700 1 _ |a Milsom, Michael D
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700 1 _ |a Harbottle, Richard
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