Journal Article

DKFZ-2021-03235

http://join2-wiki.gsi.de/foswiki/pub/Main/Artwork/join2_logo100x88.png An episomal DNA vector platform for the persistent genetic modification of pluripotent stem cells and their differentiated progeny.

Roig-Merino, A. (First author)DKFZ* ; Urban, M. (First author)DKFZ* ; Bozza, M.DKFZ* ; Peterson, J. D.DKFZ* ; Bullen, L. ; Büchler-Schäff, M.DKFZ* ; Stäble, S.DKFZ* ; van der Hoeven, F.DKFZ* ; Müller-Decker, K.DKFZ* ; McKay, T. R. ; Milsom, M. D.DKFZ* ; Harbottle, R. (Last author)DKFZ*

2022
Elsevier [New York, NY]

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Please use a persistent id in citations: doi:10.1016/j.stemcr.2021.11.011

Abstract: 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.

Keyword(s): DNA vector ; SMAR ; differentiation ; embryonic stem cell ; episome ; induced pluripotent stem cell ; nonintegrative ; reprogramming ; self-renewal ; transgenesis

Note: #EA:F160#LA:F160# /2022 Jan 11;17(1):143-158

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Contributing Institute(s):

1. F160 DNA-Vektoren (F160)
2. A012 Experimentelle Hämatologie (A012)
3. Translationale Medizinische Onkologie (B340)
4. W450 Transgen-Service (W450)
5. W420 Tumormodelle (W420)

Research Program(s):

1. 316 - Infektionen, Entzündung und Krebs (POF4-316) (POF4-316)

Appears in the scientific report 2021

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Database coverage:
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