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  4. Lentiviral vector transduction of NOD/SCID repopulating cells results in multiple vector integrations per transduced cell: risk of insertional mutagenesis
 
research article

Lentiviral vector transduction of NOD/SCID repopulating cells results in multiple vector integrations per transduced cell: risk of insertional mutagenesis

Woods, Niels-Bjarne
•
Muessig, Arne
•
Schmidt, Manfred
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2003
Blood

Efficient vector transduction of hematopoietic stem cells is a requirement for successful gene therapy of hematologic disorders. We asked whether human umbilical cord blood CD34(+)CD38(lo) nonobese diabetic/severe combined immunodeficiency (NOD/SCID) repopulating cells (SRCs) could be efficiently transduced using lentiviral vectors, with a particular focus on the average number of vector copies integrating into these primitive progenitor cells. Mouse bone marrow was analyzed by fluorescence-activated cell-sorter scanner and by semiquantitative polymerase chain reaction (PCR) to determine the transduction efficiency into SRCs. Lentiviral vector transduction resulted in an average of 22% (range, 3%-90%) of the human cells expressing green fluorescent protein (GFP), however, multiple vector copies were present in human hematopoietic cells, with an average of 5.6 +/- 3.3 (n = 12) copies per transduced cell. To confirm the ability of lentiviral vectors to integrate multiple vector copies into SRCs, linear amplification mediated (LAM)-PCR was used to analyze the integration site profile of a selected mouse showing low-level engraftment and virtually all human cells expressing GFP. Individually picked granulocyte macrophage colony-forming unit colonies derived from the bone marrow of this mouse were analyzed and shown to have the same 5 vector integrants within each colony. Interestingly, one integration site of the 5 that were sequenced in this mouse was located in a known tumor-suppressor gene, BRCA1. Therefore, these findings demonstrate the ability of lentiviral vectors to transduce multiple copies into a subset of NOD/SCID repopulating cells. While this is efficient in terms of transduction and transgene expression, it may increase the risk of insertional mutagenesis.

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Type
research article
DOI
10.1182/blood-2002-07-2238
Author(s)
Woods, Niels-Bjarne
Muessig, Arne
Schmidt, Manfred
Flygare, Johan
Olsson, Karin
Salmon, Patrick
Trono, Didier  
von Kalle, Christof
Karlsson, Stefan
Date Issued

2003

Published in
Blood
Volume

101

Issue

4

Start page

1284

End page

9

Subjects

Genetic Vectors

•

Mutagenesis

•

Insertional

•

Transfection

•

Virus Integration

Editorial or Peer reviewed

REVIEWED

Written at

EPFL

EPFL units
LVG  
Available on Infoscience
September 5, 2005
Use this identifier to reference this record
https://infoscience.epfl.ch/handle/20.500.14299/215893
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