The Origin and Development of Genetic Therapies

From Magic and Superstition to Molecular Genetics and the Human Genome
Erscheinungsjahr 2025
ISBN: 978-0-19-768997-4
Verlag: Oxford University Press

Buch, Englisch, 216 Seiten, Format (B × H): 160 mm x 234 mm, Gewicht: 318 g

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.

Autoren/Hrsg.

Friedmann, Theodore

Inhaltsverzeichnis

- 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

Über Autor(innen)

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.