Exploring the effect of sequence length and composition on allele-selective inhibition of human huntingtin expression by single-stranded silencing RNAs

Nucleic Acid Therapeutics

Volumn 24, Issue 3, 2014, Pages 199-209

  Hu, Jiaxin ^a   Liu, Jing ^a   Yu, Dongbo ^a   Aiba, Yuichiro ^a   Lee, Suheung ^a   Pendergraff, Hannah ^a,b   Boubaker, Jihane ^c   Artates, Jonathan W ^c   Lagier Tourenne, Clotilde ^c   Lima, Walt F ^d   Swayze, Eric E ^d   Prakash, Thazha P ^d   Corey, David R ^a  

^a UNIVERSITY OF TEXAS SOUTHWESTERN MEDICAL CENTER   (United States)

^b UNIVERSITY OF SOUTHAMPTON   (United Kingdom)

^c UNIVERSITY OF CALIFORNIA   (United States)

^d ISIS PHARMACEUTICALS   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

HUNTINGTIN; LIPID; MUTANT PROTEIN; OLIGONUCLEOTIDE; SINGLE STRANDED RNA;

ALLELE; ANIMAL EXPERIMENT; ARTICLE; BRAIN; CAG REPEAT; CHEMICAL MODIFICATION; FIBROBLAST CULTURE; GENE SILENCING; HUMAN; HUMAN CELL; HUNTINGTON CHOREA; MOUSE; NEUROLOGIC DISEASE; NONHUMAN; PRIORITY JOURNAL; PROTEIN EXPRESSION; RNA STRUCTURE; WESTERN BLOTTING;

ALLELES; ANIMALS; BASE SEQUENCE; BRAIN; CELL LINE; DISEASE MODELS, ANIMAL; FIBROBLASTS; GENE EXPRESSION REGULATION; HUMANS; HUNTINGTON DISEASE; INJECTIONS, INTRAVENTRICULAR; LIPIDS; MICE; MOLECULAR SEQUENCE DATA; NERVE TISSUE PROTEINS; RNA INTERFERENCE; RNA, MESSENGER; RNA, SMALL INTERFERING; STRUCTURE-ACTIVITY RELATIONSHIP;

EID: 84900842932     PISSN: 21593337     EISSN: 21593345     Source Type: Journal    
DOI: 10.1089/nat.2013.0476     Document Type: Article

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