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  4. Lentiviral vector-mediated genetic modification of human neural progenitor cells for ex vivo gene therapy
 
research article

Lentiviral vector-mediated genetic modification of human neural progenitor cells for ex vivo gene therapy

Capowski, E. E.
•
Schneider, B. L.
•
Ebert, A. D.
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2007
Journal of Neuroscience Methods

Human neural progenitor cells (hNPC) hold great potential as an ex vivo system for delivery of therapeutic proteins to the central nervous system. When cultured as aggregates, termed neurospheres, hNPC are capable of significant in vitro expansion. In the current study, we present a robust method for lentiviral vector-mediated gene delivery into hNPC that maintains the differentiation and proliferative properties of neurosphere cultures while minimizing the amount of viral vector used and controlling the number of insertion sites per population. This method results in long-term, stable expression even after differentiation of the hNPC to neurons and astrocytes and allows for generation of equivalent transgenic populations of hNPC. In addition, the in vitro analysis presented predicts the behavior of transgenic lines in vivo when transplanted into a rodent model of Parkinson's disease. The methods presented provide a powerful tool for assessing the impact of factors such as promoter systems or different transgenes on the therapeutic utility of these cells.

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Type
research article
DOI
10.1016/j.jneumeth.2007.02.022
Web of Science ID

WOS:000247945800016

Author(s)
Capowski, E. E.
Schneider, B. L.
Ebert, A. D.
Seehus, C. R.
Szulc, J.  
Zufferey, R.  
Aebischer, P.  
Svendsen, C. N.
Date Issued

2007

Published in
Journal of Neuroscience Methods
Volume

163

Issue

2

Start page

338

End page

49

Subjects

Animals

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Astrocytes/metabolism/virology

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Biological Markers/metabolism

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Brain Tissue Transplantation/methods

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Cell Differentiation/genetics

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Cell Proliferation

•

Cells

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Cultured

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Fetus

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Gene Therapy/*methods

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Gene Transfer Techniques/*standards

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Genetic Vectors/*genetics

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Glial Cell Line-Derived Neurotrophic

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Factor/biosynthesis/genetics

•

Humans

•

Lentivirus/*genetics

•

Neurons/metabolism/virology

•

Parkinson Disease/genetics/therapy

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Promoter Regions (Genetics)/genetics

•

Rats

•

Rats

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Inbred Lew

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Spheroids

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Cellular/cytology/physiology/virology

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Stem Cells/*metabolism/virology

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Transgenes

•

Rats

Note

Stem Cell Research Program, Waisman Center, University of Wisconsin-Madison, 1500 Highland Ave, Madison, WI 53705, USA. capowski@waisman.wisc.edu

Editorial or Peer reviewed

REVIEWED

Written at

EPFL

EPFL units
LEN  
Available on Infoscience
August 27, 2008
Use this identifier to reference this record
https://infoscience.epfl.ch/handle/20.500.14299/27558
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