Huntingtin haplotypes provide prioritized target panels for allele-specific silencing in huntington disease patients of european ancestry

Molecular Therapy

Volumn 23, Issue 11, 2015, Pages 1759-1771

  Kay, Chris ^a   Collins, Jennifer A ^a   Skotte, Niels H ^a   Southwell, Amber L ^a   Warby, Simon C ^b   Caron, Nicholas S ^a   Doty, Crystal N ^a   Nguyen, Betty ^a   Griguoli, Annamaria ^c   Ross, Colin J ^a   Squitieri, Ferdinando ^c   Hayden, Michael R ^a  

^a UNIVERSITY OF BRITISH COLUMBIA   (Canada)

^b UNIVERSITÉ DE MONTRÉAL   (Canada)

^c IRCCS NEUROMED   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; HUNTINGTIN; MESSENGER RNA; HTT PROTEIN, HUMAN; NERVE PROTEIN;

ALLELE; ARTICLE; CAG REPEAT; CANADA; CONTROLLED STUDY; EUROPEAN; FRANCE; GENE LOCUS; GENE MUTATION; GENE SEQUENCE; GENE TARGETING; GENETIC POLYMORPHISM; GENOTYPE; HAPLOTYPE; HETEROZYGOSITY; HUMAN; HUNTINGTON CHOREA; ITALY; MAJOR CLINICAL STUDY; MONOGENIC DISORDER; PROTEIN EXPRESSION; SINGLE NUCLEOTIDE POLYMORPHISM; SWEDEN; WILD TYPE; CAUCASIAN; GENE EXPRESSION; GENE THERAPY; GENETICS; HETEROZYGOTE; HUNTINGTON DISEASE; INDEL MUTATION; PROCEDURES; TRINUCLEOTIDE REPEAT;

ALLELES; EUROPEAN CONTINENTAL ANCESTRY GROUP; GENE EXPRESSION; GENE TARGETING; GENETIC THERAPY; HAPLOTYPES; HETEROZYGOTE; HUMANS; HUNTINGTIN PROTEIN; HUNTINGTON DISEASE; INDEL MUTATION; NERVE TISSUE PROTEINS; OLIGONUCLEOTIDES, ANTISENSE; POLYMORPHISM, SINGLE NUCLEOTIDE; RNA, MESSENGER; TRINUCLEOTIDE REPEAT EXPANSION;

EID: 84948717105     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2015.128     Document Type: Article

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