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Viral Vectors for Gene Therapy

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Biopharmaceutical Drug Design and Development

Abstract

During the past few years, substantial progress has been made in determining the cause of many of the more than 4000 human genetic diseases described to date. With the advent of the Human Genome Project and the commitment to sequence the entire human genome, a tremendous amount of data has and will continue to be generated that will be used to combating genetic diseases, infectious diseases, and cancer. Along with an increased understanding of the underlying genetic causes of specific human diseases comes a desire to permanently or temporarily correct these conditions by introducing normal genes into the malfunctioning cells of patients, and to overexpress or alter the expression of specific genes for therapeutic purposes. Certain viruses, such as retrovirus and adenovirus, have become very important as vectors for the delivery of recombinant DNA into human cells. Retrovirus is more established as a gene therapy vector up to this point; adenovirus is a relative newcomer. Although adenovirus is being considered as a vector for treating specific genetic diseases, such as cystic fibrosis, the major application may be in cancer gene therapy, because of the transient nature of adenovirus gene expression. This review will focus primarily on retrovirus and adenovirus technology, and on recent developments in how to make recombinant virus in the laboratory. The molecular biology of retroviruses and adenoviruses as it applies to gene delivery, and the status of various clinical trials using these and other virus vectors, will be discussed.

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Authors
  1. Kristoffer Valerie
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Editors and Affiliations

  1. Department of Pharmacy and Pharmaceutics, Virginia Commonwealth University, Richmond, VA, USA

    Susanna Wu-Pong PhD

  2. School of Pharmacy, West Virginia University, Morgantown, WV, USA

    Yongyut Rojanasakul PhD

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© 1999 Springer Science+Business Media New York

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Valerie, K. (1999). Viral Vectors for Gene Therapy. In: Wu-Pong, S., Rojanasakul, Y. (eds) Biopharmaceutical Drug Design and Development. Humana Press, Totowa, NJ. https://doi.org/10.1007/978-1-59259-705-5_5

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  • DOI: https://doi.org/10.1007/978-1-59259-705-5_5

  • Publisher Name: Humana Press, Totowa, NJ

  • Print ISBN: 978-1-4757-4644-0

  • Online ISBN: 978-1-59259-705-5

  • eBook Packages: Springer Book Archive

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