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Prolonged correction of hyperlipidemia in mice with familial hypercholesterolemia using an adeno-associated viral vector expressing very-low-density lipoprotein receptor.

Authors: Chen, SJ  Rader, DJ  Tazelaar, J  Kawashiri, M  Gao, G  Wilson, JM 
Citation: Chen SJ, etal., Mol Ther. 2000 Sep;2(3):256-61.
Pubmed: (View Article at PubMed) PMID:10985956
DOI: Full-text: DOI:10.1006/mthe.2000.0122

Adeno-associated viral vectors were used to deliver the gene for very-low-density lipoprotein (VLDL) receptor (VLDLR) to liver of a murine model of familial hypercholesterolemia (FH). Infusion of adeno-associated virus-VLDLR into the portal circulation of FH mice resulted in a 40% reduction in serum cholesterol and triglyceride that was stable for the duration of the study (30 weeks). Fractionation of serum lipids revealed a reduction of both VLDL and low-density lipoprotein. Expression of transgene-derived VLDLR was confirmed in livers of recipient animals by Western blot analysis and immunohistochemistry; vector DNA was present at 1 copy/cell. Vector-treated animals had significantly less lipid accumulation in liver and reduced atherosclerosis in the aorta.


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CRRD Object Information
CRRD ID: 1625570
Created: 2007-06-14
Species: All species
Last Modified: 2007-06-14
Status: ACTIVE


RGD is funded by grant HL64541 from the National Heart, Lung, and Blood Institute on behalf of the NIH.