Silencing mutant ATXN3 expression resolves molecular phenotypes in SCA3 transgenic mice

Molecular Therapy

Volumn 21, Issue 10, 2013, Pages 1909-1918

  Rodríguez Lebrón, Edgardo ^a   Costa, Maria Docarmo ^b   Luna Cancalon, Katiuska ^b   Peron, Therese M ^b   Fischer, Svetlana ^b   Boudreau, Ryan L ^a   Davidson, Beverly L ^a   Paulson, Henry L ^b  

^a UNIVERSITY OF IOWA   (United States)

^b UNIVERSITY OF MICHIGAN MEDICAL SCHOOL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ATAXIN 3; MICRORNA; PARVOVIRUS VECTOR; RNA;

3' UNTRANSLATED REGION; ADENO ASSOCIATED VIRUS; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; ATXN3 GENE; CEREBELLUM; DEGENERATIVE DISEASE; GENE; GENE EXPRESSION; GENE SILENCING; GENE TARGETING; HUMAN; HUMAN CELL; IN VIVO STUDY; MOLECULAR GENETICS; MOUSE; NEUROPATHOLOGY; NONHUMAN; PHENOTYPE; PROTEIN SYNTHESIS INHIBITION; RNA INTERFERENCE; SPINOCEREBELLAR ATAXIA TYPE 3; SPINOCEREBELLAR DEGENERATION; TRANSGENIC MOUSE; VIRAL GENE DELIVERY SYSTEM;

ADENO-ASSOCIATED VIRUS; MUS; MUS MUSCULUS;

3' UNTRANSLATED REGIONS; ANIMALS; CEREBELLUM; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; GENE EXPRESSION REGULATION; GENE SILENCING; GENETIC VECTORS; HEK293 CELLS; HUMANS; MACHADO-JOSEPH DISEASE; MICE; MICE, TRANSGENIC; MICRORNAS; MOLECULAR MIMICRY; MOLECULAR TARGETED THERAPY; MUTANT PROTEINS; NERVE TISSUE PROTEINS; NUCLEAR PROTEINS; PHENOTYPE; REPRESSOR PROTEINS; TRANSDUCTION, GENETIC;

EID: 84885023525     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2013.152     Document Type: Article

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