This book is a collection of preclinical and clinical reports on the application of gene therapy to human disease. The two methods available for delivering therapeutic genes to diseased cells, viral and non-viral vector systems, are detailed and characterized. Several reports describe both approaches currently used in gene therapy: the introduction of a wild-type gene to restore normal gene function in diseased cells, and the use of antisense molecules to hinder abnormal gene expression. Clinical studies detail several different strategies for the treatment of cancer and cardiovascular diseases, using genetic material as therapeutic agents. The regulatory issues governing the use of gene therapy in Europe and the United States are also presented. This book highlights the range of applications and demonstrates the rapid progress being made in the field of gene therapy.
