From the discoveries of the principles of genetic inheritance in the 19th century to the development of the concepts of human molecular genetic disease in the 20th century, the treatment of human disease has been transformed from a focus on symptoms to the goal of correcting underlying genetic defects which are the cause of many human diseases. Here, Theodore Friedmann shows that this scientific revolution in medicine compares in importance to Copernicus's demonstration of the sun-centered order of the planetary system, the 18th and 19th century discoveries of the cellular structure of living systems, germ theory of disease, the invention of anaesthesia, and the discovery that genetic information is stored and transmitted by DNA. The Origin and Development of Genetic Therapies explores the origins and early developmental history of the medical field of gene therapy. Friedmann examines the early failures and increasingly promising clinical ""successes"" for this new approach to cure genetic disease and explores how the concepts of gene therapy can affect human hereditary and human evolution.
Introduction 1. Early Western concepts of disease and therapy 2. From Galen to the Renaissance - anatomy and the cell and germ theories 3. Darwin, Mendel, and Galton - the discovery, disappearance, and rediscovery of the laws of inheritance 4. The rediscovery of Mendel, Garrod, and human biochemical genetics 5. Mutations are inherited by Mendel's laws and can cause disease. What are chromosomes? What elements in chromosomes cause disease? 6. DNA Is the repository and transmitter of genetic information 7. From inborn errors to molecular disease 8. First faltering steps towards gene therapy - viruses as gene transfer vectors 9. Birth of molecular biology and recombinant DNA - the remarkable 1960s-1970s 10. Potential misstep becomes reality - the emergence of federal oversight and regulation 11. Oversight and regulation of recombinant DNA research -- the Asilomar Conferences 12. Chemical non-viral vectors 13. Genetics - from a descriptive to a manipulative science 14. Early clinical gene therapy trials 15. From academia to the bedside - the design of clinical trials 16. The Human Genome Project - a complement, but not the origin, of gene therapy 17. A third serious setback 18. Finally - break-through success? 19. Gene editing - a foundational new era for genetic therapies 20. RNA-based therapies and programmable RNA editing 21. The role of biotech and pharma in the development of gene therapy 22. Current dilemma and future directions 23. Summary: genetic therapies - a new field of medicine
Theodore Friedmann completed his medical training at the University of Pennsylvania, and clinical training in Pediatrics at the Boston Children's Hospital. He was awarded postdoctoral fellowships at the universities of Cambridge and Oxford, the U.S. National Institutes of Health, and the Salk Institute. In 1971, he joined the pediatric faculty of the School of Medicine at the University of California San Diego where he is currently affiliated as Emeritus Professor of Pediatrics.
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