The Origin and Development of Genetic Therapies

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