Gene Therapy

Mauro Giacca, MD, PhD, is the Director of the Italian Component of the International Centre for Genetic Engineering and Biotechnology (ICGEB), an international research organisation based in Trieste, Italy, and full professor of molecular biology at the Faculty of Medicine of the University of Trieste. Over the last 20 years, he has participated in the front line in the development of the gene therapy field. He is internationally recognized as an expert in AAV vector biology and in the application of AAV vectors for the gene therapy of cardiovascular disorders.

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.