RGD Reference Report - Prolonged correction of hyperlipidemia in mice with familial hypercholesterolemia using an adeno-associated viral vector expressing very-low-density lipoprotein receptor. - Rat Genome Database

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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.
RGD ID: 1625570
Pubmed: PMID:10985956   (View Abstract at PubMed)
DOI: DOI:10.1006/mthe.2000.0122   (Journal Full-text)

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.

RGD Manual Disease Annotations    Click to see Annotation Detail View

Objects Annotated

Genes (Rattus norvegicus)
Vldlr  (very low density lipoprotein receptor)

Genes (Mus musculus)
Vldlr  (very low density lipoprotein receptor)

Genes (Homo sapiens)
VLDLR  (very low density lipoprotein receptor)


Additional Information