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Viral Vectors for Gene Therapy pp 3–26Cite as

Basic Concepts in Viral Vector-Mediated Gene Therapy

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Part of the book series: Methods in Molecular Biology ((MIMB,volume 1937))

Abstract

Today any researcher with the desire can easily purchase a viral vector. However, despite the availability of viral vectors themselves, the requisite knowledge that is absolutely essential to conducting a gene therapy experiment remains somewhat obscure and esoteric. To utilize viral vectors to their full potential, a large number of decisions must be made, in some instances prior to even obtaining the vector itself. For example, critical decisions include selection of the proper virus, selection of the proper expression cassette, whether to produce or purchase a viral vector, proper viral handling and storage, the most appropriate delivery method, selecting the proper controls, how to ensure your virus is expressing properly, and many other complex decisions that are essential to performing a successful gene therapy experiment. The need to make so many important decisions can be overwhelming and potentially prohibitive, especially to the novice gene therapist. In order to aid in this challenging process, here we provide an overview of basic gene therapy modalities and a decision tree that can be used to make oneself aware of the options available to the beginning gene therapist. This information can be used as a road map to help navigate the complex and perhaps confusing process of designing a successful gene therapy experiment.

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Authors and Affiliations

  1. Department of Translational Science and Molecular Medicine, Michigan State University, Grand Rapids, MI, USA

    Matthew J. Benskey, Ivette M. Sandoval, Kathryn Miller, Rhyomi L. Sellnow, Aysegul Gezer, Nathan C. Kuhn, Roslyn Vashon & Fredric P. Manfredsson

  2. Mercy Health Saint Mary’s, Grand Rapids, MI, USA

    Ivette M. Sandoval & Fredric P. Manfredsson

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  1. Matthew J. Benskey
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  2. Ivette M. Sandoval
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  3. Kathryn Miller
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  4. Rhyomi L. Sellnow
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  5. Aysegul Gezer
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  6. Nathan C. Kuhn
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  7. Roslyn Vashon
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  8. Fredric P. Manfredsson
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Correspondence to Fredric P. Manfredsson .

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  1. Translational Science and Molecular Medicine, Michigan State University, Grand Rapids, MI, USA

    Fredric P. Manfredsson

  2. Translational Science and Molecular Medicine, Michigan State University, Grand Rapids, MI, USA

    Matthew J. Benskey

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Benskey, M.J. et al. (2019). Basic Concepts in Viral Vector-Mediated Gene Therapy. In: Manfredsson, F., Benskey, M. (eds) Viral Vectors for Gene Therapy. Methods in Molecular Biology, vol 1937. Humana Press, New York, NY. https://doi.org/10.1007/978-1-4939-9065-8_1

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