Ataxin-3 protein modification as a treatment strategy for spinocerebellar ataxia type 3: Removal of the CAG containing exon

Neurobiology of Disease

Volumn 58, Issue , 2013, Pages 49-56

  Evers, Melvin M ^a   Tran, Hoang Dai ^a   Zalachoras, Ioannis ^a   Pepers, Barry A ^a   Meijer, Onno C ^a   den Dunnen, Johan T ^a   van Ommen, Gert Jan B ^a   Aartsma Rus, Annemieke ^a   van Roon Mom, Willeke M C ^a  

^a LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

Author keywords

Ataxin 3; CAG repeat; Exon skipping; Polyglutamine disorder; Polyglutamine repeat; Spinocerebellar ataxia type 3

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; ATAXIN 3; MUTANT PROTEIN; POLYGLUTAMINE; UBIQUITIN;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; BRAIN REGION; CAG REPEAT; CELL DEATH; CEREBELLUM; CONTROLLED STUDY; DRUG DESIGN; DRUG MECHANISM; EXON SKIPPING; HUMAN; HUMAN CELL; IN VITRO STUDY; IN VIVO STUDY; MALE; MOUSE; NEUROTOXICITY; NONHUMAN; PRIORITY JOURNAL; PROTEIN BINDING; PROTEIN FUNCTION; PROTEIN MODIFICATION; SINGLE DRUG DOSE; SPINOCEREBELLAR ATAXIA TYPE 3; SPINOCEREBELLAR DEGENERATION; WILD TYPE;

ANTISENSE OLIGONUCLEOTIDE; AON; ATAXIN-3; ATXN3; CAG REPEAT; DMD; DUCHENNE MUSCULAR DYSTROPHY; EXON SKIPPING; ICV; INTRA-CEREBRAL VENTRICULAR; MACHADO-JOSEPH DISEASE; MJD; NES; NLS; NUCLEAR EXPORT SIGNAL; NUCLEAR LOCALIZATION SIGNAL; POLYGLUTAMINE; POLYGLUTAMINE DISORDER; POLYGLUTAMINE REPEAT; POLYQ; RNA INTERFERENCE; RNAI; SCA3; SINGLE NUCLEOTIDE POLYMORPHISM; SNP; SPINOCEREBELLAR ATAXIA TYPE 3; UBIQUITIN INTERACTING MOTIFS; UIMS; VALOSIN CONTAINING PROTEIN; VCP;

ANIMALS; CELLS, CULTURED; DNA MUTATIONAL ANALYSIS; DOSE-RESPONSE RELATIONSHIP, DRUG; EXONS; FIBROBLASTS; HUMANS; MACHADO-JOSEPH DISEASE; MICE; MICE, INBRED C57BL; MUTATION; NERVE TISSUE PROTEINS; NUCLEAR PROTEINS; OLIGONUCLEOTIDES, ANTISENSE; PEPTIDES; PROTEIN BINDING; REPRESSOR PROTEINS; RNA, MESSENGER; TRANSFECTION; TRINUCLEOTIDE REPEATS; UBIQUITIN;

EID: 84879377492     PISSN: 09699961     EISSN: 1095953X     Source Type: Journal    
DOI: 10.1016/j.nbd.2013.04.019     Document Type: Article

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