Sleeping beauty transposon vectors in liver-directed gene delivery of LDLR and VLDLR for gene therapy of familial hypercholesterolemia

Molecular Therapy

Volumn 24, Issue 3, 2016, Pages 620-635

  Turunen, Tytteli A K ^a   Kurkipuro, Jere ^a   Heikura, Tommi ^a   Vuorio, Taina ^a   Hytönen, Elisa ^a   Izsvák, Zsuzsanna ^b   Ylä Herttuala, Seppo ^a,c  

^a UNIVERSITY OF EASTERN FINLAND   (Finland)

^b MAX DELBRÜCK CENTER FOR MOLECULAR MEDICINE   (Germany)

^c KUOPIO UNIVERSITY HOSPITAL   (Finland)

Author keywords

[No Author keywords available]

Indexed keywords

BETA GALACTOSIDASE; CHOLESTEROL; LOW DENSITY LIPOPROTEIN RECEPTOR; PLASMID VECTOR; VERY LOW DENSITY LIPOPROTEIN RECEPTOR; LIPID; TRANSPOSON;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; AORTA ATHEROSCLEROSIS; ARTICLE; CHOLESTEROL BLOOD LEVEL; CHOLESTEROL LIVER LEVEL; CONTROLLED STUDY; DRUG MECHANISM; DRUG SCREENING; ELECTROPORATION; FAMILIAL HYPERCHOLESTEROLEMIA; FATTY LIVER; FEMALE; FOLLOW UP; GENE DOSAGE; GENE EXPRESSION; HISTOPATHOLOGY; HUMAN; HUMAN CELL; HYDRODYNAMICS BASED TRANSFECTION; IN VITRO STUDY; IN VIVO STUDY; LACZ GENE; LDLR GENE; LIPID STORAGE; LIVER FUNCTION; LIVER STRUCTURE; MALE; MOUSE; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; NONVIRAL GENE THERAPY; SLEEPING BEAUTY TRANSPOSON; TRANSGENE; TRANSPOSON; VLDLR GENE; ANIMAL; AORTA; ATHEROSCLEROSIS; BLOOD; CELL LINE; DISEASE MODEL; GENE ORDER; GENE THERAPY; GENE TRANSFER; GENE VECTOR; GENETICS; HYPERLIPOPROTEINEMIA TYPE II; KNOCKOUT MOUSE; LIVER; METABOLISM; PATHOLOGY;

ANIMALS; AORTA; ATHEROSCLEROSIS; CELL LINE; DISEASE MODELS, ANIMAL; DNA TRANSPOSABLE ELEMENTS; GENE EXPRESSION; GENE ORDER; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; GENETIC VECTORS; HYPERLIPOPROTEINEMIA TYPE II; LIPIDS; LIVER; MICE; MICE, KNOCKOUT; RECEPTORS, LDL;

EID: 84960394482     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2015.221     Document Type: Article

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