The expanded CAG repeat in the huntingtin gene as target for therapeutic RNA modulation throughout the HD mouse brain

PLoS ONE

Volumn 12, Issue 2, 2017, Pages

  Datson, Nicole A ^a   González Barriga, Anchel ^a   Kourkouta, Eleni ^a   Weij, Rudie ^a   Van De Giessen, Jeroen ^a   Mulders, Susan ^a   Kontkanen, Outi ^b   Heikkinen, Taneli ^b   Lehtimäki, Kimmo ^b   Van Deutekom, Judith C T ^a  

^a BIOMARIN PHARMACEUTICAL INC   (United States)

^b Charles River Discovery Research Services   (Finland)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; ANTISENSE OLIGONUCLEOTIDE CUG 7; DRUG VEHICLE; HUNTINGTIN; INOSITOL; MESSENGER RNA; N ACETYLASPARTIC ACID; PHOSPHOPROTEIN DARPP 32; UNCLASSIFIED DRUG; COMPLEMENTARY RNA; HTT PROTEIN, MOUSE;

AMINO TERMINAL SEQUENCE; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTISENSE THERAPY; ARTICLE; BRAIN CORTEX; BRAIN SIZE; CAG REPEAT; CONTROLLED STUDY; CORPUS STRIATUM; DARPP 32 GENE; DRUG MEGADOSE; FEMALE; GAIT; GENE; GENE TARGETING; GRIP STRENGTH TEST; HIPPOCAMPUS; HTT GENE; HUNTINGTON CHOREA; IN VIVO STUDY; LOW DRUG DOSE; MALE; MOTOR ACTIVITY; MOTOR PERFORMANCE; MOUSE; NEUROANATOMY; NEUROCHEMISTRY; NEUROMUSCULAR FUNCTION; NONHUMAN; OPEN FIELD TEST; ROTAROD TEST; TREATMENT DURATION; ANIMAL; BRAIN; C57BL MOUSE; GENE THERAPY; GENETICS; GLIOSIS; HUNTINGTON DISEASE; METABOLISM; PATHOLOGY; TRINUCLEOTIDE REPEAT;

ANIMALS; BRAIN; FEMALE; GENETIC THERAPY; GLIOSIS; HUNTINGTIN PROTEIN; HUNTINGTON DISEASE; MALE; MICE; MICE, INBRED C57BL; MOTOR ACTIVITY; RNA, ANTISENSE; TRINUCLEOTIDE REPEAT EXPANSION;

EID: 85012075676     PISSN: None     EISSN: 19326203     Source Type: Journal    
DOI: 10.1371/journal.pone.0171127     Document Type: Article

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