Exon skipping of hepatic APOB pre-mRNA with splice-switching oligonucleotides reduces LDL cholesterol In Vivo

Molecular Therapy

Volumn 21, Issue 3, 2013, Pages 602-609

  Disterer, Petra ^a   Al Shawi, Raya ^a   Ellmerich, Stephan ^a   Waddington, Simon N ^a,b   Owen, James S ^a   Simons, J Paul ^a   Khoo, Bernard ^a,c  

^a UNIVERSITY COLLEGE LONDON   (United Kingdom)

^b UNIVERSITY OF THE WITWATERSRAND   (South Africa)

^c ROYAL FREE AND UNIVERSITY COLLEGE MEDICAL SCHOOL   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

ALANINE AMINOTRANSFERASE; ANTILIPEMIC AGENT; APOLIPOPROTEIN B; APOLIPOPROTEIN B87; ASPARTATE AMINOTRANSFERASE; HYPOCHOLESTEROLEMIC AGENT; ISOPROTEIN; LOW DENSITY LIPOPROTEIN; LOW DENSITY LIPOPROTEIN CHOLESTEROL; MESSENGER RNA; OLIGONUCLEOTIDE; PRAVASTATIN; SPLICE SWITCHING OLIGONUCLEOTIDE; SPLICE SWITCHING OLIGONUCLEOTIDE 1 PF; SPLICE SWITCHING OLIGONUCLEOTIDE 14 53P; SPLICE SWITCHING OLIGONUCLEOTIDE 14 53PF; SPLICE SWITCHING OLIGONUCLEOTIDE 14 P; SPLICE SWITCHING OLIGONUCLEOTIDE 14 PF; UNCLASSIFIED DRUG; VERY LOW DENSITY LIPOPROTEIN;

ANIMAL EXPERIMENT; ARTICLE; CELL ASSAY; CELL STRAIN CACO 2; CELL STRAIN HEPG2; CHOLESTEROL BLOOD LEVEL; CONTROLLED STUDY; DOSE TIME EFFECT RELATION; DRUG DOSE COMPARISON; DRUG DOSE ESCALATION; DRUG EFFECT; EXON SKIPPING; FAMILIAL HYPOBETALIPOPROTEINEMIA; HUMAN; HUMAN CELL; IN VITRO STUDY; IN VIVO STUDY; LIPOPROTEIN SYNTHESIS; MOUSE; NONHUMAN; PROTEIN ASSEMBLY; PROTEIN EXPRESSION; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; SINGLE DRUG DOSE; STEROL ANALYSIS; UPREGULATION; WESTERN BLOTTING;

MUS;

EID: 84875227516     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2012.264     Document Type: Article

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