Chronicles the origins and early developmental history of the new medical field of gene therapy. Friedmann examines the early failures and increasingly promising clinical "successes" for this new approach to cure genetic disease.
Chronicles the origins and early developmental history of the new medical field of gene therapy. Friedmann examines the early failures and increasingly promising clinical "successes" for this new approach to cure genetic disease.
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.
Inhaltsangabe
* 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
* 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
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