AAV vectors expressing LDLR gain-of-function variants demonstrate increased efficacy in mouse models of familial hypercholesterolemia

Circulation Research

Volumn 115, Issue 6, 2014, Pages 591-599

  Somanathan, Suryanarayan ^a   Jacobs, Frank ^a,b   Wang, Qiang ^a   Hanlon, Alexandra L ^a   Wilson, James M ^a   Rader, Daniel J ^a  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b JANSSEN RESEARCH FOUNDATION   (Belgium)

Author keywords

genetic therapy; human; hyperlipoproteinemia type II; low density lipoprotein receptor; PCSK9 protein

Indexed keywords

APOLIPOPROTEIN B MESSENGER RNA EDITING ENZYME CATALYTIC POLYPEPTIDE 1; COMPLEMENTARY DNA; LOW DENSITY LIPOPROTEIN RECEPTOR; MESSENGER RNA; PARVOVIRUS VECTOR; PROPROTEIN CONVERTASE KEXIN TYPE 9; SERINE PROTEINASE; UNCLASSIFIED DRUG;

AMINO ACID SUBSTITUTION; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CHOLESTEROL BLOOD LEVEL; FAMILIAL HYPERCHOLESTEROLEMIA; GAIN OF FUNCTION MUTATION; GENE EXPRESSION; GENE THERAPY; GENETIC VARIABILITY; HEK293 CELL LINE; IN VITRO STUDY; KNOCKOUT MOUSE; MOUSE; NONHUMAN; PRIORITY JOURNAL; PROTEIN DEGRADATION; RECEPTOR DOWN REGULATION; TRANSGENE;

GENETIC THERAPY; HYPERLIPOPROTEINEMIA TYPE II; LOW-DENSITY LIPOPROTEIN RECEPTOR, HUMAN; PCSK9 PROTEIN, HUMAN;

ANIMALS; CHOLESTEROL; CYTIDINE DEAMINASE; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; GENETIC THERAPY; GENETIC VARIATION; GENETIC VECTORS; HEK293 CELLS; HUMANS; HYPERLIPOPROTEINEMIA TYPE II; MALE; MICE; MICE, KNOCKOUT; PROPROTEIN CONVERTASES; RECEPTORS, LDL; SERINE ENDOPEPTIDASES; TREATMENT OUTCOME;

EID: 84907140899     PISSN: 00097330     EISSN: 15244571     Source Type: Journal    
DOI: 10.1161/CIRCRESAHA.115.304008     Document Type: Article

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