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Development of gene therapy for hematopoietic stem cells using lentiviral vectors.

Woods, Niels-Bjarne LU ; Ooka, Andreas LU and Karlsson, Stefan LU (2002) In Leukemia 16(4). p.563-569
Abstract
Lentiviral vectors are promising tools for the development of gene therapy since they can transduce both quiescent and dividing target cells. Lentiviral vectors may be particularly promising gene delivery tools for hematopoietic stem cells since these target cells tend to be quiescent and are therefore difficult target cells for vectors that require dividing targets. Human hematopoietic stem cells that can repopulate NOD/SCID mice have been efficiently transduced using HIV-1-based lentiviral vectors and similar vectors can also transduce murine hematopoietic stem cells. HIV-1 vectors that contain strong general promoters can generate high levels of transgene expression and very high expression levels can be generated in erythroid cells in... (More)
Lentiviral vectors are promising tools for the development of gene therapy since they can transduce both quiescent and dividing target cells. Lentiviral vectors may be particularly promising gene delivery tools for hematopoietic stem cells since these target cells tend to be quiescent and are therefore difficult target cells for vectors that require dividing targets. Human hematopoietic stem cells that can repopulate NOD/SCID mice have been efficiently transduced using HIV-1-based lentiviral vectors and similar vectors can also transduce murine hematopoietic stem cells. HIV-1 vectors that contain strong general promoters can generate high levels of transgene expression and very high expression levels can be generated in erythroid cells in vivo using beta-globin regulatory sequences to control the expression of the transgene. Current lentiviral vectors have a similar level of biosafety as oncoretroviral vectors and can therefore theoretically be used in clinical gene therapy protocols. Future challenges include the generation of lentiviral vectors that can express more than one transgene at high levels and the generation of safe permanent packaging cells for practical use in clinical gene therapy trials. (Less)
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author
organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
Lentivirus : genetics, Human, Genetic Vectors, Hematopoietic Stem Cells : metabolism, Animal, Gene Therapy, Leukemia : therapy, Leukemia : metabolism
in
Leukemia
volume
16
issue
4
pages
563 - 569
publisher
Nature Publishing Group
external identifiers
  • pmid:11960333
  • wos:000175193000015
  • scopus:0036242306
ISSN
1476-5551
DOI
10.1038/sj/leu/2402447
language
English
LU publication?
yes
id
2e6cb33b-d6d0-430b-a8d6-372159374f65 (old id 107723)
alternative location
http://www.nature.com/leu/journal/v16/n4/abs/2402447a.html
date added to LUP
2007-07-11 13:34:31
date last changed
2017-10-08 04:11:46
@article{2e6cb33b-d6d0-430b-a8d6-372159374f65,
  abstract     = {Lentiviral vectors are promising tools for the development of gene therapy since they can transduce both quiescent and dividing target cells. Lentiviral vectors may be particularly promising gene delivery tools for hematopoietic stem cells since these target cells tend to be quiescent and are therefore difficult target cells for vectors that require dividing targets. Human hematopoietic stem cells that can repopulate NOD/SCID mice have been efficiently transduced using HIV-1-based lentiviral vectors and similar vectors can also transduce murine hematopoietic stem cells. HIV-1 vectors that contain strong general promoters can generate high levels of transgene expression and very high expression levels can be generated in erythroid cells in vivo using beta-globin regulatory sequences to control the expression of the transgene. Current lentiviral vectors have a similar level of biosafety as oncoretroviral vectors and can therefore theoretically be used in clinical gene therapy protocols. Future challenges include the generation of lentiviral vectors that can express more than one transgene at high levels and the generation of safe permanent packaging cells for practical use in clinical gene therapy trials.},
  author       = {Woods, Niels-Bjarne and Ooka, Andreas and Karlsson, Stefan},
  issn         = {1476-5551},
  keyword      = {Lentivirus : genetics,Human,Genetic Vectors,Hematopoietic Stem Cells : metabolism,Animal,Gene Therapy,Leukemia : therapy,Leukemia : metabolism},
  language     = {eng},
  number       = {4},
  pages        = {563--569},
  publisher    = {Nature Publishing Group},
  series       = {Leukemia},
  title        = {Development of gene therapy for hematopoietic stem cells using lentiviral vectors.},
  url          = {http://dx.doi.org/10.1038/sj/leu/2402447},
  volume       = {16},
  year         = {2002},
}