Basis for Gene Therapy

During the last quarter century, there has been an explosion of reports in the scientific literature about genetic engineering to transfer or alter genes and use the information and methodology for the therapy of human disease. Until recently, curricula have included limited information about molecular genetics, and many clinicians, investigators, and students have found publications about clinical trials and research in molecular genetics difficult to follow. In response, this text presents in clear and understandable form the current status of medical molecular and genetic engineering. Major subjects discussed include: the eukaryotic cell, deoxyribonucleic acid, mitochondria, replicons, structure of genes, gene transfer, genetic engineering, transcription and translation, the immune system, clones, stem cells and vectors, genomic probes, clinical genetics, mutations, inherited diseases, chromosomes, oncogenes and tumor-suppressor genes, human gene therapy, programmed cell death in gene therapy, P53 tumor-suppressor gene, synthetic gene delivery, suicide gene prodrug therapy, immunotherapy for cancer, vaccines, gene transfer, and models for gene therapy. The book has been written in a manner to extend its usefulness by including a list of internet resources in numerous web sites; a glossary including acronyms; and a list of vendors providing kits, supplies, and useful protocols for molecular genetics research and clinical trials. After reading this text, the reader should be able to explore the applicable literature with increased ability to understand gene therapy terminology and the basic research supporting clinical trials. The book will be extremely useful to clinicians and others in managing the medical care of their patients and in following the literature related to the progress that will undoubtedly lead to a golden age of medicine. For example, the most recent reports of the sequence of base pairs in the entire human genome appears in the February 2, 2001 issue of Science.