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Origin and Development of Genetic Therapies: From Magic and Superstition to Molecular Genetics and the Human Genome [Pehme köide]

(Emeritus Professor of Pediatrics, UCSD School of Medicine)
  • Formaat: Paperback / softback, 216 pages, kõrgus x laius x paksus: 234x160x18 mm, kaal: 318 g, 113 halftones
  • Ilmumisaeg: 02-Oct-2025
  • Kirjastus: Oxford University Press Inc
  • ISBN-10: 0197689973
  • ISBN-13: 9780197689974
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  • Formaat: Paperback / softback, 216 pages, kõrgus x laius x paksus: 234x160x18 mm, kaal: 318 g, 113 halftones
  • Ilmumisaeg: 02-Oct-2025
  • Kirjastus: Oxford University Press Inc
  • ISBN-10: 0197689973
  • ISBN-13: 9780197689974
Teised raamatud teemal:
"Abstract. This book is intended to examine the origins and development of the revolutionary new medical field of gene therapy. Understanding the history of gene therapy depends on an appreciation of the concept of disease, how it evolved in response to advances in science and Medicine from ancient cultures to modern times and, in particular, to the birth of human genetics. Central to that history has been the revelation that most human disease does not reflect magical or mystical actions of aggrieved deities but rather results from interactions of natural worldly factors with inherent normal and aberrant features of human biology. That knowledge identified foreign natural factors such as microbial agents responsible and that, in turn, led to discovery of methods such as vaccines and antibiotics to interfere with these exogenous disease-causing agents. Remarkably, Medicine has now developed tools and methods to control and alter the inherent factors that define both normal and aberrant human biology - i.e., genes, and made those factors suitable targets for therapy. Key words. Ancient concepts of health, disease and therapy, advances in medical sciences, Aesculapeus, Hippocrates, Galen, religion, aggrieved deities, communion with nature"--

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.

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.