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The only new gene therapy textbook of the last 8 years.
Presents a very broad and comprehensive book written by a specialist in the field.
Includes many illustrations and schemes in color.
This book presents to the reader a comprehensive and detailed, yet easily accessible, description of the whole field of gene therapy. It covers topics ranging from vector development to the results of the most recent clinical trials. It includes descriptions of lentiviral and AAV vector development, of therapeutic gene selection (including siRNAs, shRNAs, and microRNAs), and of the most recent clinical applications of gene therapy for diseases of the eye, the cardiovascular system, and the central nervous system. Of note, the book only considers conditions for which transfer of genetic material has already progressed to clinical applications or is close to doing so. The language is 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 fields, whenever technical descriptions are required these are provided. Readers will include graduate students in the life sciences area in their final years of study, PhD students, and medical doctors specializing in different fields of internal and specialist medicine, in addition to research scientists working in various areas of current biomedical research.
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.