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Navigable Slide Index

1. Introduction
2. Outline (1)
3. Lipoprotein lipase biology
4. Consequences of loss of LPL activity
5. Genetic LPL deficiency
6. Clinical phenotype of LPL deficiency
7. Gene therapy for LPL deficient patients
8. Outline (2)
9. Gene replacement
10. Vector - adeno-associated virus
11. Transgene - LPLS447X
12. AMT-010 vector
13. Vector production
14. Proof of principle in LPL deficient mice
15. Proof of principle in LPL deficient mice - long term
16. AMT010 in LPL deficient mice - dose dependency
17. Feline LPL deficiency
18. Vector administration
19. Proof of principle in LPL deficient cats
20. LPL deficient cats - antibodies development
21. LPL deficient cats - immunosuppression (1)
22. LPL deficient cats - immunosuppression (2)
23. Gene delivery to myoblasts
24. Infection of myoblasts with AMT010
25. Conclusions for clinical trial
26. Outline (3)
27. Premonitoring study (1)
28. Premonitoring study (2)
29. Clinical trial
30. Clinical trial - design & inclusion criteria
31. Clinical trial - baseline characteristics
32. Clinical trial - gene defects and baseline
33. Administration of AMT-010
34. Clinical trial - safety analyses
35. Aknowledgements

Topics Covered

• No treatment for genetic lipoprotein lipase (LPL) deficiency
• This disorder has several specific characteristics which make it a feasible target for gene therapy approaches
• Development of LPL transgene delivery
• Use of adeno-associated virus

Links

Series:

• Genetics of Cardiovascular Disease

Categories:

• Clinical Practice
• Pharmaceutical Sciences

Therapeutic Areas:

• Cardiovascular & Metabolic

Talk Citation

Kuivenhoven, J.A. (2008, November 24). Gene therapy as a therapeutic option for lipoprotein lipase deficiency [Video file]. In The Biomedical & Life Sciences Collection, Henry Stewart Talks. Retrieved December 21, 2024, from https://doi.org/10.69645/UGZH8204.
Export Citation (RIS)

Publication History

• Published on November 24, 2008
• Archived on February 25, 2021

Financial Disclosures

• Dr. Jan Albert Kuivenhoven has not informed HSTalks of any commercial/financial relationship that it is appropriate to disclose.