Abstract
The development of in vivo gene transfer technology has created a powerful new tool for the study of diseases by providing methods to overexpress or to inhibit specific local factors that are believed to contribute to a pathological process. In addition, this technology provides the opportunity for the development of novel therapeutic strategies such as gene replacement, gene correction, or gene augmentation paving the way for gene therapy as a therapeutic option for many diseases (1,2). The recombinant DNA “breakthrough” has provided us with a new and powerful approach to the questions that have intrigued and plagued humans for centuries (3). This paradigmatic shift in medicine led to a change of the view of pathophysiology from a more biochemical interpretation to the recognition of disease as a molecular event on the level of gene expression (4,5). New therapeutic approaches are moving from biochemically designed pharmaceuticals to genetically engineered tools for the treatment of diseases. The elucidation of molecular and cellular pathobiological processes of diseases has depended on (1) in vitro cell culture experiments, (2) studies of gene expression in experimental animal models or human specimens using Northern blot, reverse transcription-polymerase chain reaction (RT-PCR), or in situ hybridization, and (3) the development of transgenic animal models and the use of homologous recombination.
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von der Leyen, H.E. (2000). Gene Therapy and Nitric Oxide. In: Loscalzo, J., Vita, J.A. (eds) Nitric Oxide and the Cardiovascular System. Contemporary Cardiology. Humana Press, Totowa, NJ. https://doi.org/10.1007/978-1-59259-002-5_28
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DOI: https://doi.org/10.1007/978-1-59259-002-5_28
Publisher Name: Humana Press, Totowa, NJ
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