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Nonviral Gene Delivery

  • Chapter
Biopharmaceutical Drug Design and Development

Abstract

Gene therapy became a clinical reality when Rosenberg et al. (1) treated two young patients with adenine deaminase (ADA) deficiency with a retroviral vector that had been biotechnologic ally manipulated to encode for human ADA. Since 1990, approx 300 clinical trials, with a total of over 2000 volunteers or patients participating, have been conducted. Viral vectors predominate over synthetic approaches, which account for approx 25% of the 2100 patients enrolled in clinical gene therapy protocols worldwide (2). Although originally conceived as a therapy for genetic diseases, i.e., replacement of a defect or missing gene, the concept of gene therapy has since been dramatically expanded to include the gene as a drug and the gene as a vaccine. Acute inflammatory diseases, such as adult respiratory distress syndrome could benefit from the temporary overexpression of genes that produce antioxidants, e.g., superoxide dismutase/catalase, or interact with the prostaglandin/leukotriene cascade (3). Transfection of cells, specifically, cancer cells, with genes that generate presentation of nonself immune-responsive moieties at the cell surface, has led to the development of DNA vaccines, to date perhaps the most successful and promising subfield within the area of gene therapy (4).

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  1. Frank L. Sorgi
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  2. Hans Schreier
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  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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Sorgi, F.L., Schreier, H. (1999). Nonviral Gene Delivery. 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_6

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