Adeno-associated virus serotype 8 gene therapy leads to significant lowering of plasma cholesterol levels in humanized mouse models of homozygous and heterozygous familial hypercholesterolemia

Human Gene Therapy

Volumn 24, Issue 1, 2013, Pages 19-26

  Kassim, Sadik H ^a   Li, Hui ^b   Bell, Peter ^a   Somanathan, Suryanarayan ^a   Lagor, William ^b   Jacobs, Frank ^a   Billheimer, Jeffrey ^b   Wilson, James M ^a   Rader, Daniel J ^b  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; APOLIPOPROTEIN B MESSENGER RNA EDITING ENZYME CATALYTIC POLYPEPTIDE 1; APOLIPOPROTEIN B100; COMPLEMENTARY DNA; INTERMEDIATE DENSITY LIPOPROTEIN; LOW DENSITY LIPOPROTEIN CHOLESTEROL; LOW DENSITY LIPOPROTEIN RECEPTOR; MESSENGER RNA; TRIACYLGLYCEROL; VERY LOW DENSITY LIPOPROTEIN;

ADENO ASSOCIATED VIRUS; ADENO ASSOCIATED VIRUS SEROTYPE 8; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CHOLESTEROL BLOOD LEVEL; CONTROLLED STUDY; FAMILIAL HYPERCHOLESTEROLEMIA; GENE THERAPY; HETEROZYGOTE; HETEROZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA; HOMOZYGOTE; HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA; MALE; MOUSE; NONHUMAN; PROTEIN EXPRESSION; PROTEIN INTERACTION; TRANSGENE; TRIACYLGLYCEROL BLOOD LEVEL;

ANIMALS; CHOLESTEROL; CYTIDINE DEAMINASE; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; ENZYME-LINKED IMMUNOSPOT ASSAY; GENETIC THERAPY; GENETIC VECTORS; HUMANS; HYPERLIPOPROTEINEMIA TYPE II; IMMUNOBLOTTING; INTERFERON-GAMMA; LIVER; MICE; MICE, INBRED C57BL; MICE, KNOCKOUT; RECEPTORS, LDL;

ADENO-ASSOCIATED VIRUS; MUS;

EID: 84872773410     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2012.108     Document Type: Article

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