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Retrovirus Vectors and Regulatable Promoters

  • Chapter
Gene Therapy for Neurological Disorders and Brain Tumors

Part of the book series: Contemporary Neuroscience ((CNEURO))

Abstract

Transfer of therapeutic genes into diseased cells using retroviral vectors is a promising approach for the treatment of certain, virtually incurable diseases. The rapid development of technology in human gene therapy has been facilitated by the use of retroviruses as gene delivery vectors. Most retroviral vectors have been derived from Moloney murine leukemia virus (MoMuLV) (1, 2). Several features of MoMuLV make it an ideal vector for use in gene therapy. Using an appropriate packaging cell line (i.e., ecotropic or amphotropic), a wide variety of cell types of both rodent and human origin can be infected with high efficiency (3–5). Viral integration is very efficient in that the structure of the viral genome is retained in the proviral DNA (the reverse transcribed DNA form of the virus) (6–8), facilitating the delivery and expression of a therapeutic gene(s). Furthermore, integration can only occur in host-cells in the process of replicating and synthesizing their own genomic DNA (an exception is in the human immunodeficiency virus [HIV] family of retroviruses), which allows the targeting of the virus to rapidly dividing cells and not postmitotic cells (9–11). The viral genes in the vector genome which encode the gag, pol, and env proteins are dispensable, provided they are replaced in trans by uniquely designed packaging cell lines; however, the iv packaging signal is needed for efficient packaging of the vector RNAs into the viral capsid. Removing these genes from the vector genome allows the production of replication-defective virus that can deliver a suitable gene to a target cell. Because the target cells lack the packaging cell functions, further spread of the virus to other tissues is effectively restricted.

Subjectively, to evolve must most often have amounted to suffering from a disease. And these diseases were of course molecular. The appearance of the concept of good and evil, interpreted by man as his painful expulsion from paradise, was probably a molecular disease that turned out to be evolution.

Zuckerkandl and Pauling, 1962

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Authors
  1. Steven A. Reeves
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  1. Molecular Neuro-Oncology, Neurosurgical Service, Massachussetts General Hospital, Harvard Medical School, Boston, MA, USA

    E. Antonio Chiocca MD, PhD

  2. Neurogenetics Unit, Massachussetts General Hospital, Harvard Medical Center, Boston, MA, USA

    Xandra O. Breakefield PhD

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Reeves, S.A. (1998). Retrovirus Vectors and Regulatable Promoters. In: Chiocca, E.A., Breakefield, X.O. (eds) Gene Therapy for Neurological Disorders and Brain Tumors. Contemporary Neuroscience. Humana Press, Totowa, NJ. https://doi.org/10.1007/978-1-59259-478-8_1

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  • DOI: https://doi.org/10.1007/978-1-59259-478-8_1

  • Publisher Name: Humana Press, Totowa, NJ

  • Print ISBN: 978-1-4757-5314-1

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