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Lentiviral Vectors Derived from Simian Immunodeficiency Virus

  • D. Nègre
  • G. Duisit
  • P.-E. Mangeot
  • P. Moullier
  • J.-L. Darlix
  • F.-L. Cosset
Chapter
Part of the Current Topics in Microbiology and Immunology book series (CT MICROBIOLOGY, volume 261)

Abstract

The ultimate success of gene therapy to cure inherited or acquired genetic diseases relies on the development and on the availability of gene transfer vectors that can efficiently deliver a transgene following their administration in vivo. Several challenging hurdles need to be overcome to reach such a goal. A first prerequisite is that methods that allow the preparation of vectors at high titers and in culture systems with potential for large scale-up need to be optimized (ANDREADIS et al. 1999; KOTANI et al. 1994; SMITH et al. 1996). Second, the gene transfer vectors should not be recognized by the host immune system in order to avoid their inactivation (COSSET et al. 1995b; DEPOLO et al. 1999). Upon their delivery into gene therapy recipients, vectors should also be able to circumvent the numerous biological barriers that are likely to limit their diffusion and bio-distribution in the target organism. They should therefore be able to specifically recognize, penetrate and express the transgene in cells of the gene therapy target tissue (DIAZ et al. 1998; JAGER et al. 1999; RUSSELL and COSSET 1999). Third, they should be able to replicate and to express a transgene in cells that are not proliferating or are slowly proliferating, a predominant situation in vivo. Last, but not least, they should be accepted by both ethical and regulatory authorities. In this respect the development of vectors derived from viruses that are not pathogenic to humans may be preferred.

Keywords

Long Terminal Repeat Transfer Vector Ciency Virus Human Gene Therapy Packaging Cell Line 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.

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Copyright information

© Springer-Verlag Berlin Heidelberg 2002

Authors and Affiliations

  • D. Nègre
    • 1
    • 4
  • G. Duisit
    • 2
  • P.-E. Mangeot
    • 3
  • P. Moullier
    • 2
  • J.-L. Darlix
    • 3
  • F.-L. Cosset
    • 1
  1. 1.Laboratoire de Vectorologie Rétrovirale et Thérapie GéniqueINSERM U412, Ecole Normale Supérieure de Lyon and IFR 74LyonFrance
  2. 2.Laboratoire de Thérapie GéniqueCHU Hotel DieuNantesFrance
  3. 3.LaboRétroLyonFrance
  4. 4.Lyon Cedex 07France

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