Pseudotyped recombinant adeno-associated viral vectors mediate efficient gene transfer into primary human CD34+ peripheral blood progenitor cells

Because of their pluripotency, human CD34+ peripheral blood progenitor cells (PBPC) are targets of interest for the treatment of many acquired and inherited disorders using gene therapeutic approaches. Unfortunately, most current vector systems lack either sufficient transduction efficiency or an ap...

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Main Authors: Veldwijk, Marlon Romano (Author) , Sellner, Leopold (Author) , Stiefelhagen, Marius (Author) , Kleinschmidt, Jürgen (Author) , Maier-Laufs, Stephanie (Author) , Topaly, Julian (Author) , Frühauf, Stefan (Author) , Zeller, W. Jens (Author) , Wenz, Frederik (Author)
Format: Article (Journal)
Language:English
Published: 2010
In: Cytotherapy
Year: 2010, Volume: 12, Issue: 1, Pages: 107-112
ISSN:1477-2566
DOI:10.3109/14653240903348293
Online Access:Resolving-System, lizenzpflichtig, Volltext: https://doi.org/10.3109/14653240903348293
Verlag, lizenzpflichtig, Volltext: https://www.sciencedirect.com/science/article/pii/S1465324910703728
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Author Notes:Marlon R. Veldwijk, Leopold Sellner, Marius Stiefelhagen, Jürgen A. Kleinschmidt, Stephanie Laufs, Julian Topaly, Stefan Fruehauf, W. Jens Zeller & Frederik Wenz
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Summary:Because of their pluripotency, human CD34+ peripheral blood progenitor cells (PBPC) are targets of interest for the treatment of many acquired and inherited disorders using gene therapeutic approaches. Unfortunately, most current vector systems lack either sufficient transduction efficiency or an appropriate safety profile. Standard single-stranded recombinant adeno-associated virus 2 (AAV2)-based vectors offer an advantageous safety profile, yet lack the required efficiency in human PBPC.
Item Description:Online veröffentlicht am 20. Dezember 2012
Gesehen am 31.08.2023
Physical Description:Online Resource
ISSN:1477-2566
DOI:10.3109/14653240903348293