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Lentivirus Gene Engineering Protocols pp 17–27Cite as

The Choice of a Suitable Lentivirus Vector

Transcriptional Targeting

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Part of the book series: Methods in Molecular Biology™ ((MIMB,volume 229))

Abstract

Human immunodeficiency virus (HIV)-derived lentiviral vectors can integrate into the genome of dividing and nondividing cells, in vitro as well as in vivo (reviewed in refs. 1–3). Lentiviral vectors are particularly efficient in transducing multipotent stem cells, such as hematopoietic stem cells (HSC), without compromising their self-renewing and organ repopulation capacity upon transplantation in vivo (4–6). This is a crucial advantage over oncoretroviral vectors derived from the Moloney murine leukemia virus (MoMLV) since transplantation of genetically modified stem cells (hematopoietic, neural, or epidermal) is a potential therapy for a variety of genetic and acquired disorders, including diabetes, multiple sclerosis, cancer, and acquired immunodeficiency syndrome (AIDS). Several years of research have drastically improved both design and packaging of lentiviral vectors, minimized the use of HIV structural and regulatory sequences in both the transfer and the packaging constructs and have virtually abolished the safety concerns originally raised by the idea of transducing human cells with a derivative of HIV (reviewed in ref. 3). These vectors are now a very promising alternative to transduce transplantable stem cells ex vivo and such postmitotic tissues as the central nervous system (CNS) or liver in vivo.

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Author information

Authors and Affiliations

  1. HSR-Telethon Institute of Gene Therapy, Istituto Scientifico H. San Raffaele, Milan, Italy

    Francesco Lotti

  2. Department of Biomedical Sciences, University of Modena School of Medicine, Modena, Italy

    Fulvio Mavilio

Authors
  1. Francesco Lotti
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  2. Fulvio Mavilio
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Editor information

Editors and Affiliations

  1. Laboratory of Virology, Istituto Superiore di Sanitá, Rome, Italy

    Maurizio Federico

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© 2003 Humana Press Inc.

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Lotti, F., Mavilio, F. (2003). The Choice of a Suitable Lentivirus Vector. In: Federico, M. (eds) Lentivirus Gene Engineering Protocols. Methods in Molecular Biology™, vol 229. Humana Press. https://doi.org/10.1385/1-59259-393-3:17

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  • DOI: https://doi.org/10.1385/1-59259-393-3:17

  • Publisher Name: Humana Press

  • Print ISBN: 978-1-58829-091-5

  • Online ISBN: 978-1-59259-393-4

  • eBook Packages: Springer Protocols

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