Abstract
Gene-based therapies are emerging as safe and effective treatments for genetic diseases. In this introductory chapter, we provide a general overview of the gene therapy principles and vectors for applications in inherited diseases. Next, we discuss some of the most relevant gene therapy clinical trials.
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Brunetti-Pierri, N. (2017). Gene Transfer Strategies and Applications in Genetic Diseases. In: Brunetti-Pierri, N. (eds) Safety and Efficacy of Gene-Based Therapeutics for Inherited Disorders. Springer, Cham. https://doi.org/10.1007/978-3-319-53457-2_1
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DOI: https://doi.org/10.1007/978-3-319-53457-2_1
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