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- Epidemiology and Risk Factors
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1. Coronary heart disease epidemiology: global context for a new genetic understanding
- Prof. Harry Hemingway
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2. Cardiovascular risk factors
- Dr. Michal Vrablik
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3. Lipoproteins
- Prof. Arnold von Eckardstein
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4. Thrombotic risk factors for cardiovascular disease
- Prof. Gordon Lowe
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5. Lipoprotein(a)
- Dr. Jaimini Cegla
- Biology of Coronary Heart Disease
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6. Plaque rupture
- Prof. Petri Kovanen
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7. Transcription factors and complex disease development
- Dr. Ines Pineda-Torra
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8. Animal models to explore cardiovascular disease
- Prof. Martin Merkel
- Treatment
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9. Diagnosis and treatment of dyslipidemias
- Prof. Anton Stalenhoef
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10. Key drug discovery challenges in cardiovascular medicine
- Dr. Dan Swerdlow
- Dr. Michael V. Holmes
- Genetics
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11. Moving from GWAS hits to functional variants
- Prof. Steve Humphries
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12. Heart disease genes and SNPs
- Prof. Steve Humphries
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13. Familial hypercholesterolaemia: genetic causes and treatment
- Prof. Steve Humphries
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14. Familial hypercholesterolaemia: cascade testing and monogenic vs. polygenic causes
- Prof. Steve Humphries
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16. Genetics of cardiovascular disease
- Prof. Philippa Talmud
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17. The genetics of CHD: moving research findings into patient benefit
- Prof. Steve Humphries
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19. The genetics of abdominal aortic aneurysm
- Dr. Seamus Harrison
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20. Genome scans for hypertension
- Prof. Patricia Munroe
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21. Telomeres and cardiovascular disease
- Dr. Jess Buxton
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23. Genetics of hypertrophic cardiomyopathy (HCM)
- Dr. Petros Syrris
- Archived Lectures *These may not cover the latest advances in the field
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24. Gene-environment interaction and oxidative stress in cardiovascular disease
- Dr. Jeffrey Stephens
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25. Gene therapy as a therapeutic option for lipoprotein lipase deficiency
- Dr. Jan Albert Kuivenhoven
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26. Cardiovascular diseases: from epidemiology to nutritional interventions
- Dr. Antonis Zampelas
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27. Genetic testing for CHD risk: fact or fiction?
- Prof. Steve Humphries
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28. Pharmacogenetics: progress, pitfalls and clinical potential
- Prof. Steve Humphries
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29. Familial hyperchlolesterolaemia: a monogenic cause of early CHD
- Prof. Steve Humphries
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30. Lipid metabolism
- Dr. Ulrike Beisiegel
Printable Handouts
Navigable Slide Index
- Introduction
- Outline (1)
- Lipoprotein lipase biology
- Consequences of loss of LPL activity
- Genetic LPL deficiency
- Clinical phenotype of LPL deficiency
- Gene therapy for LPL deficient patients
- Outline (2)
- Gene replacement
- Vector - adeno-associated virus
- Transgene - LPLS447X
- AMT-010 vector
- Vector production
- Proof of principle in LPL deficient mice
- Proof of principle in LPL deficient mice - long term
- AMT010 in LPL deficient mice - dose dependency
- Feline LPL deficiency
- Vector administration
- Proof of principle in LPL deficient cats
- LPL deficient cats - antibodies development
- LPL deficient cats - immunosuppression (1)
- LPL deficient cats - immunosuppression (2)
- Gene delivery to myoblasts
- Infection of myoblasts with AMT010
- Conclusions for clinical trial
- Outline (3)
- Premonitoring study (1)
- Premonitoring study (2)
- Clinical trial
- Clinical trial - design & inclusion criteria
- Clinical trial - baseline characteristics
- Clinical trial - gene defects and baseline
- Administration of AMT-010
- Clinical trial - safety analyses
- 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:
Categories:
Therapeutic Areas:
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 March 21, 2025, from https://doi.org/10.69645/UGZH8204.Export Citation (RIS)
Publication History
Financial Disclosures
- Dr. Jan Albert Kuivenhoven has not informed HSTalks of any commercial/financial relationship that it is appropriate to disclose.
Gene therapy as a therapeutic option for lipoprotein lipase deficiency
A selection of talks on Cardiovascular & Metabolic
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