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@ARTICLE{RoigMerino:178288,
author = {A. Roig-Merino$^*$ and M. Urban$^*$ and M. Bozza$^*$ and J.
D. Peterson$^*$ and L. Bullen and M. Büchler-Schäff$^*$
and S. Stäble$^*$ and F. van der Hoeven$^*$ and K.
Müller-Decker$^*$ and T. R. McKay and M. D. Milsom$^*$ and
R. Harbottle$^*$},
title = {{A}n episomal {DNA} vector platform for the persistent
genetic modification of pluripotent stem cells and their
differentiated progeny.},
journal = {Stem cell reports},
volume = {17},
number = {1},
issn = {2213-6711},
address = {[New York, NY]},
publisher = {Elsevier},
reportid = {DKFZ-2021-03235},
pages = {143-158},
year = {2022},
note = {#EA:F160#LA:F160# /2022 Jan 11;17(1):143-158},
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.},
keywords = {DNA vector (Other) / SMAR (Other) / differentiation (Other)
/ embryonic stem cell (Other) / episome (Other) / induced
pluripotent stem cell (Other) / nonintegrative (Other) /
reprogramming (Other) / self-renewal (Other) / transgenesis
(Other)},
cin = {F160 / A012 / B340 / W450 / W420},
ddc = {610},
cid = {I:(DE-He78)F160-20160331 / I:(DE-He78)A012-20160331 /
I:(DE-He78)B340-20160331 / I:(DE-He78)W450-20160331 /
I:(DE-He78)W420-20160331},
pnm = {316 - Infektionen, Entzündung und Krebs (POF4-316)},
pid = {G:(DE-HGF)POF4-316},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:34942088},
doi = {10.1016/j.stemcr.2021.11.011},
url = {https://inrepo02.dkfz.de/record/178288},
}