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I entered the gene therapy field in the mid-1990s, being fascinated by the immense potential of genes as drugs for the treatment of human disease. Since then, I have experienced the ups and downs of this discipline, and tried to contribute with my work and that of my laboratory to the development of innovative approaches to the treatment of cardiovascular disorders. During these years, I have had several opp- tunities to speak on gene therapy at lectures and academic lessons, and have often noticed that the field is very attractive to scientists of all disciplines. However, as yet no comprehensive book on the subject has been published. Indeed, most books in the field are either a collection of gene transfer laboratory protocols or deal with the subject in a rather superficial manner. Hence the idea to write a gene therapy textbook that is broad and comprehensive, but at the same time provides sufficient molecular and clinical detail to be of interest to students, professors, and specialists in the various disciplines that contribute to gene therapy. I have tried to keep the language plain and, whenever possible, non-technical. Since the book is intended to be a textbook in the field of gene therapy in both the basic science and clinical areas, whenever technical descriptions are required, they are provided.

Gene Therapy

Abbreviations.- 1 Introduction to Gene Therapy .- 1.1 Genes as Drugs .- 1.2 Gene Therapy: An Overview .- Further Reading .- 2 Therapeutic Nucleic Acids .- 2.1 Protein-Coding Genes .- 2.2 Non-Coding Nucleic Acids.- Further Reading .- Selected Bibliography.- 3 Methods for Gene Delivery.- 3.1 Cellular Barriers to Gene Delivery.- 3.2 Direct Inoculation of DNAs and RNAs.- 3.3 Physical Methods.- 3.4 Chemical Methods.- 3.5 Viral Vectors.- Further Reading.- Selected Bibliography.- 4 Clinical Applications of Gene Therapy .- 4.1 Clinical Applications of Gene Therapy: General Considerations.- 4.2 Gene Therapy of Hematopoietic Stem Cells.- 4.3 Gene Therapy of Cystic Fibrosis.- 4.4 Gene Therapy of Muscular Dystrophies.- 4.5 Gene Therapy of Hemophilia.- 4.6 Gene Therapy of Cancer.- 4.7 Gene Therapy of Neurodegenerative Disorders.- 4.8 Gene Therapy of Eye Diseases.- 4.9 Gene Therapy of Cardiovascular Disorders.- 4.10 Gene Therapy of HIV Infection.- 4.11 Gene Therapy of Liver Diseases.- Further Reading.- Selected Bibliography.- 5 Ethical and Social Problems of Gene Therapy.- 5.1 Safety of Clinical Experimentation.- 5.2 Gene Therapy of Germ Cells.- 5.3 In Utero Gene Therapy.- 5.4 Gene Therapy of the Embryo.- 5.5 Gene Transfer for Cosmetic Appearance and Gene Doping.- Further Reading.- Selected Bibliography.- Subject Index.