Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterol

Nature Biotechnology

Volumn 36, Issue 8, 2018, Pages 717-725

  Wang, Lili ^a   Smith, Jeff ^b   Breton, Camilo ^a   Clark, Peter ^a   Zhang, Jia ^a   Ying, Lei ^a   Che, Yan ^a   Lape, Janel ^b   Bell, Peter ^a   Calcedo, Roberto ^a   Buza, Elizabeth L ^a   Saveliev, Alexei ^a   Bartsevich, Victor V ^b   He, Zhenning ^a   White, John ^a   Li, Mingyao ^c   Jantz, Derek ^b   Wilson, James M ^a  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b PRECISION BIOSCIENCES   (United States)

^c UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

MAMMALS; VIRUSES;

ADENO-ASSOCIATED VIRUS VECTORS; CLINICAL TRANSLATION; HUMAN GENETIC DISEASE; HYPERCHOLESTEROLEMIA; NON-HUMAN PRIMATE; PRECLINICAL STUDIES; SAFETY CONSIDERATIONS; SECOND GENERATION;

CHOLESTEROL;

ADENO ASSOCIATED VIRUS VECTOR; AMINOTRANSFERASE; ENDONUCLEASE; LOW DENSITY LIPOPROTEIN CHOLESTEROL; MEGANUCLEASE; PROPROTEIN CONVERTASE 9; UNCLASSIFIED DRUG; CHOLESTEROL; DEOXYRIBONUCLEASE; LOW DENSITY LIPOPROTEIN RECEPTOR;

AMINOTRANSFERASE BLOOD LEVEL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CELL POPULATION; CHOLESTEROL BLOOD LEVEL; CONTROLLED STUDY; ENZYME INACTIVATION; FEMALE; GENE EDITING; GENOME; HUMAN; HUMAN CELL; IMMUNOTOXICITY; INDUCED PLURIPOTENT STEM CELL; LIVER; LIVER BIOPSY; LIVER CELL; MACACA; MALE; NONHUMAN; PRIORITY JOURNAL; PROTEIN CLEAVAGE; PROTEIN EXPRESSION; PROTEIN TARGETING; T LYMPHOCYTE; TRANSGENE; ANIMAL; ANTAGONISTS AND INHIBITORS; BLOOD; DEPENDOPARVOVIRUS; ENZYMOLOGY; GENE VECTOR; GENETICS; HEK293 CELL LINE; HYPERCHOLESTEROLEMIA; KNOCKOUT MOUSE; METABOLISM; MOUSE; RHESUS MONKEY;

ANIMALS; CHOLESTEROL; DEOXYRIBONUCLEASES; DEPENDOVIRUS; GENE EDITING; GENETIC VECTORS; HEK293 CELLS; HEPATOCYTES; HUMANS; HYPERCHOLESTEROLEMIA; INDUCED PLURIPOTENT STEM CELLS; LIVER; MACACA MULATTA; MALE; MICE; MICE, KNOCKOUT; PROPROTEIN CONVERTASE 9; RECEPTORS, LDL;

EID: 85051820108     PISSN: 10870156     EISSN: 15461696     Source Type: Journal    
DOI: 10.1038/nbt.4182     Document Type: Article

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