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Navigable Slide Index

1. Introduction
2. Treatment of human disease
3. Gregor Mendel - 1865
4. Archibald Garrod 1908
5. Mutation, disease & therapeutic approaches
6. DNA structure discovery
7. Can DNA be a therapeutic agent?
8. Lesch-Nyhan disease
9. Metabolic pathway of Lesch-Nyhan disease
10. Can we modify cells with foreign DNA? (1)
11. Can we modify cells with foreign DNA? (2)
12. HPRT(-) cells can't incorporate hypoxanthine
13. Transient HPRT expression after transfection
14. Viruses as vectors
15. Converting normal cells into tumor cells with a virus
16. 1972 - human gene therapy paper
17. Gene transfer by retrovirus
18. Retroviral vector corrects a disease phenotype
19. Different vectors for transferring DNA into cells
20. Extensive regulation and oversight
21. Optimism about human gene therapy (1)
22. Optimism about human gene therapy (2)
23. A difficult controversial beginning
24. Criticism against human gene therapy
25. Gene transfer trials by year
26. Death from gene therapy trial - 1999 (1)
27. Death from gene therapy trial - 1999 (2)
28. Severe combined immunodeficiency diseases
29. Texas bubble boy
30. SCID-X1; Gamma-c deficiency
31. Success, but at a cost
32. A vector integrated foreign DNA into the lmo2 gene
33. Leber's congenital amaurosis
34. Why do children with Leber's not see?
35. rRPE65 defect in a dog model
36. Sub-retinal injection of AAV2.hRPE65v2
37. Gene therapy for Leber's amaurosis
38. Summary - the reality of gene therapy

Topics Covered

• Background in genetic concepts
• Application by Sir Archibald Garrod to human disease
• Discovery of the structure of DNA and new opportunities to understand and manipulate genes
• Proof of principle of gene transfer into human cells using the Lesch Nyhan disease model
• Rapid expansion of clinical gene therapy trials, high level of optimism and expectation
• Major setback in human OTC gene therapy study
• Patient death
• Scientific and clinical success with another disease model
• Severe combined immunodeficiency (SCID)
• Setback in SCID
• Leukemia in some treated patients
• Additional clinical successes, especially in Leber's congenital blindness

Links

Series:

• Introduction to Human Genetics

Categories:

• Clinical Practice
• Genetics & Epigenetics
• Methods

Talk Citation

Friedmann, T. (2010, October 18). The development and evolution of human gene therapy [Video file]. In The Biomedical & Life Sciences Collection, Henry Stewart Talks. Retrieved April 15, 2025, from https://doi.org/10.69645/EMRY7540.
Export Citation (RIS)

Publication History

• Published on October 18, 2010
• Archived on September 8, 2024

Financial Disclosures

• Prof. Theodore Friedmann has not informed HSTalks of any commercial/financial relationship that it is appropriate to disclose.