In silico and in vitro evaluation of exonic and intronic off-target effects form a critical element of therapeutic ASO gapmer optimization

Nucleic Acids Research

Volumn 43, Issue 18, 2015, Pages 8638-8650

  Kamola, Piotr J ^a,b,c   Kitson, Jeremy D A ^b   Turner, Gemma ^b,f   Maratou, Klio ^b   Eriksson, Sofie ^b,g   Panjwani, Aliza ^b   Warnock, Linda C ^b   Douillard Guilloux, Gaelle A ^d   Moores, Kitty ^b   Koppe, Emma L ^b   Wixted, William E ^e   Wilson, Paul A ^b   Gooderham, Nigel J ^a   Gant, Timothy W ^c   Clark, Kenneth L ^b   Hughes, Stephen A ^b   Edbrooke, Mark R ^b   Parry, Joel D ^b  

^a IMPERIAL COLLEGE LONDON   (United Kingdom)

^b GLAXOSMITHKLINE   (United Kingdom)

^c PUBLIC HEALTH ENGLAND   (United Kingdom)

^d GLAXOSMITHKLINE   (France)

^e GLAXOSMITHKLINE   (United States)

^f UNIVERSITY OF GLASGOW   (United Kingdom)

^g THE FRANCIS CRICK INSTITUTE   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; LOCKED NUCLEIC ACID; MESSENGER RNA; PHOSPHOROTHIOIC ACID; RIBONUCLEASE H; RIBONUCLEASE HI;

ARTICLE; BINDING AFFINITY; CELL NUCLEUS; COMPLEMENT ACTIVATION; CONTROLLED STUDY; CYTOPLASM; ENZYME ACTIVITY; EXON; GENE SILENCING; HUMAN; HUMAN CELL; HYBRIDIZATION; IN VITRO STUDY; INTRON; MISMATCH REPAIR; NUCLEOTIDE SEQUENCE; OFF TARGET EFFECT; POLYMERASE CHAIN REACTION; PREDICTION; PRIORITY JOURNAL; PROCESS OPTIMIZATION; PROTEIN CLEAVAGE; QUANTITATIVE ANALYSIS; SEQUENCE ALIGNMENT; WESTERN BLOTTING; BASE MISPAIRING; CELL CULTURE; CHEMISTRY; COMPUTER SIMULATION; METABOLISM;

BASE PAIR MISMATCH; CELLS, CULTURED; COMPUTER SIMULATION; EXONS; GENE KNOCKDOWN TECHNIQUES; GENE SILENCING; HUMANS; INTRONS; OLIGONUCLEOTIDES, ANTISENSE; RIBONUCLEASE H;

EID: 84949911119     PISSN: 03051048     EISSN: 13624962     Source Type: Journal    
DOI: 10.1093/nar/gkv857     Document Type: Article

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