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This book gathers international experts and leaders in different vector platforms to review the state of the art advances on gene transfer vectors, their disease applications, and their toxicity profiles. The different approaches described in each chapter take into consideration the type of vector, the biomedical knowledge of the disease targets and address the complexities and toxicology of gene delivery. Some of the strategies have already been tested in the clinic but much more needs to be learned about long-term toxicity of gene transfer strategies. This book will provide a comprehensive and authoritative overview for drug discovery researchers, toxicologists, pharmacologists, molecular biologists, physicians and students in these fields.