Silencing mutant ATXN3 expression resolves molecular phenotypes in SCA3 transgenic mice.

Rodríguez-Lebrón, Edgardo; Costa, Maria do Carmo; Costa, Maria doCarmo; Luna-Cancalon, Katiuska; Peron, Therese M; Fischer, Svetlana; Boudreau, Ryan L; Davidson, Beverly L; Paulson, Henry L

Rodríguez-Lebrón, Edgardo; Costa, Maria do Carmo; Costa, Maria doCarmo; Luna-Cancalon, Katiuska; Peron, Therese M; Fischer, Svetlana; Boudreau, Ryan L; Davidson, Beverly L; Paulson, Henry L.

Affiliation

Rodríguez-Lebrón E; Department of Internal Medicine, University of Iowa, Iowa City, Iowa, USA.

Mol Ther ; 21(10): 1909-18, 2013 Oct.

Article in En | MEDLINE | ID: mdl-23820820

Subject(s)

Machado-Joseph Disease/pathology; MicroRNAs/adverse effects; Mutant Proteins/drug effects; Nerve Tissue Proteins/drug effects; Nuclear Proteins/drug effects; Phenotype; Repressor Proteins/drug effects; 3' Untranslated Regions; Animals; Ataxin-3; Cerebellum/pathology; Dependovirus/drug effects; Dependovirus/genetics; Disease Models, Animal; Gene Expression Regulation; Gene Silencing; Genetic Vectors/drug effects; Genetic Vectors/genetics; HEK293 Cells; Humans; Machado-Joseph Disease/genetics; Mice; Mice, Transgenic; MicroRNAs/pharmacology; Molecular Mimicry; Molecular Targeted Therapy; Nerve Tissue Proteins/genetics; Nerve Tissue Proteins/metabolism; Nuclear Proteins/genetics; Nuclear Proteins/metabolism; Repressor Proteins/genetics; Repressor Proteins/metabolism; Transduction, Genetic/methods

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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Phenotype / Repressor Proteins / Nuclear Proteins / Machado-Joseph Disease / MicroRNAs / Mutant Proteins / Nerve Tissue Proteins Type of study: Prognostic_studies Limits: Animals / Humans Language: En Journal: Mol Ther Journal subject: BIOLOGIA MOLECULAR / TERAPEUTICA Year: 2013 Type: Article Affiliation country: United States

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