Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: Providing a therapeutic option for all Huntington disease patients

PLoS ONE

Volumn 9, Issue 9, 2014, Pages

  Skotte, Niels H ^a   Southwell, Amber L ^a   Østergaard, Michael E ^b   Carroll, Jeffrey B ^c   Warby, Simon C ^d   Doty, Crystal N ^a   Petoukhov, Eugenia ^a   Vaid, Kuljeet ^a   Kordasiewicz, Holly ^b   Watt, Andrew T ^b   Freier, Susan M ^b   Hung, Gene ^b   Seth, Punit P ^b   Bennett, C Frank ^b   Swayze, Eric E ^b   Hayden, Michael R ^a  

^a UNIVERSITY OF BRITISH COLUMBIA   (Canada)

^b ISIS PHARMACEUTICALS   (United States)

^c WESTERN WASHINGTON UNIVERSITY   (United States)

^d UNIVERSITÉ DE MONTRÉAL   (Canada)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; HUNTINGTIN; SPECTRIN; HTT PROTEIN, HUMAN; NERVE PROTEIN;

ANIMAL CELL; ARTICLE; BRAIN CELL CULTURE; CHEMICAL MODIFICATION; CONTROLLED STUDY; EMBRYO; FIBROBLAST; GENE SILENCING; HUMAN; HUMAN CELL; HUNTINGTON CHOREA; MOUSE; NONHUMAN; SINGLE NUCLEOTIDE POLYMORPHISM; STRUCTURE ACTIVITY RELATION; ALLELE; ANIMAL; ANIMAL EMBRYO; ANTAGONISTS AND INHIBITORS; CHEMISTRY; DRUG DESIGN; FEMALE; GENE EXPRESSION; GENE THERAPY; GENETICS; HIPPOCAMPUS; HUNTINGTON DISEASE; METABOLISM; MOLECULAR GENETICS; MUTATION; NERVE CELL; NUCLEOTIDE SEQUENCE; PATHOLOGY; PRIMARY CELL CULTURE; RNA INTERFERENCE;

ALLELES; ANIMALS; BASE SEQUENCE; DRUG DESIGN; EMBRYO, MAMMALIAN; FEMALE; GENE EXPRESSION; GENETIC THERAPY; HIPPOCAMPUS; HUMANS; HUNTINGTON DISEASE; MICE; MOLECULAR SEQUENCE DATA; MUTATION; NERVE TISSUE PROTEINS; NEURONS; OLIGONUCLEOTIDES, ANTISENSE; POLYMORPHISM, SINGLE NUCLEOTIDE; PRIMARY CELL CULTURE; RNA INTERFERENCE; STRUCTURE-ACTIVITY RELATIONSHIP;

EID: 84926687571     PISSN: None     EISSN: 19326203     Source Type: Journal    
DOI: 10.1371/journal.pone.0107434     Document Type: Article

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