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The aim of this book is to cover key aspects of existing problems in the field of development and future perspectives in gene therapy. Contributions consist of basic and translational research, as well as clinical experiences, and they outline functional mechanisms, predictive approaches, patient-related studies and upcoming challenges in this stimulating but also controversial field of gene therapy research. This source will make our doctors become comfortable with the common problems of gene therapy and inspire others to delve a bit more deeply into a topic of interest.