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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 December 14, 2024, 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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