Antisense Oligonucleotide-Mediated Removal of the Polyglutamine Repeat in Spinocerebellar Ataxia Type 3 Mice

Molecular Therapy Nucleic Acids

Volumn 8, Issue , 2017, Pages 232-242

  Toonen, Lodewijk J A ^a   Rigo, Frank ^b   van Attikum, Haico ^a   van Roon Mom, Willeke M C ^a  

^a LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

^b ISIS PHARMACEUTICALS   (United States)

Author keywords

antisense oligonucleotides; ataxin 3; ATXN3; exon skipping; Machado Joseph disease; SCA3; spinocerebellar ataxia type 3

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; ATAXIN 3; MESSENGER RNA; POLYGLUTAMINE; UBIQUITIN;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CONTROLLED STUDY; EXON; HUMAN; HUMAN CELL; MOUSE; NONHUMAN; PRIORITY JOURNAL; PROTEIN BINDING; PROTEIN DEGRADATION; PROTEIN FUNCTION; PROTEIN MODIFICATION; RNA SPLICING; SIGNAL TRANSDUCTION; SPINOCEREBELLAR DEGENERATION; UBIQUITINATION;

EID: 85029307856     PISSN: None     EISSN: 21622531     Source Type: Journal    
DOI: 10.1016/j.omtn.2017.06.019     Document Type: Article

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