Abstract
Retroviral vectors are optimal gene transfer vehicles for gene therapy, since they integrate into the target cell genome and therefore can provide permanent expression of a therapeutic gene. This is a particularly desired feature in gene therapy of monogenic inherited diseases, in which the ultimate goal is a single gene transfer event that would lead to life-long correction of the genetic defect. Moreover, retroviral vectors are thoroughly characterized, and their extensive use in preclinical and clinical studies has verified their efficacy and safety.
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Wahlfors, J., Morgan, R.A. (2002). Minigene-Containing Retroviral Vectors Using an Alphavirus/Retrovirus Hybrid Vector System. In: Morgan, J.R. (eds) Gene Therapy Protocols. Methods in Molecular Medicine, vol 69. Springer, Totowa, NJ. https://doi.org/10.1385/1-59259-141-8:173
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DOI: https://doi.org/10.1385/1-59259-141-8:173
Publisher Name: Springer, Totowa, NJ
Print ISBN: 978-0-89603-723-6
Online ISBN: 978-1-59259-141-1
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