Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse Models

Molecular Therapy Nucleic Acids

Volumn 7, Issue , 2017, Pages 200-210

  Moore, Lauren R ^a   Rajpal, Gautam ^a   Dillingham, Ian T ^a   Qutob, Maya ^a   Blumenstein, Kate G ^a   Gattis, Danielle ^b   Hung, Gene ^b   Kordasiewicz, Holly B ^b   Paulson, Henry L ^a   McLoughlin, Hayley S ^a  

^a UNIVERSITY OF MICHIGAN MEDICAL SCHOOL   (United States)

^b ISIS PHARMACEUTICALS   (United States)

Author keywords

antisense oligonucleotide; ASO; ATXN3; Machado Joseph disease; MJD; polyglutamine disease; SCA3; spinocerebellar ataxia type 3

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; ATAXIN 3; COMPLEMENTARY DNA; MUTANT PROTEIN; POLYGLUTAMINE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; ATXN3 GENE; BRAIN REGION; CAG REPEAT; CEREBELLUM; CERVICAL SPINAL CORD; CONTROLLED STUDY; CYTOMEGALOVIRUS; DIENCEPHALON; DRUG TARGETING; EVALUATION STUDY; FOREBRAIN; GENE; GENE EXPRESSION; GENE SILENCING; HUMAN; HUMAN CELL; IMMUNOFLUORESCENCE; MOUSE; NONHUMAN; PRIORITY JOURNAL; SPINOCEREBELLAR ATAXIA TYPE 3; SPINOCEREBELLAR DEGENERATION; TRANSGENE; YEAST ARTIFICIAL CHROMOSOME;

EID: 85020707985     PISSN: None     EISSN: 21622531     Source Type: Journal    
DOI: 10.1016/j.omtn.2017.04.005     Document Type: Article

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