Silencing mutant ATXN3 expression resolves molecular phenotypes in SCA3 transgenic mice. [electronic resource]
Producer: 20140429Description: 1909-18 p. digitalISSN:- 1525-0024
- 3' Untranslated Regions
- Animals
- Ataxin-3
- Cerebellum -- pathology
- Dependovirus -- drug effects
- Disease Models, Animal
- Gene Expression Regulation
- Gene Silencing
- Genetic Vectors -- drug effects
- HEK293 Cells
- Humans
- Machado-Joseph Disease -- genetics
- Mice
- Mice, Transgenic
- MicroRNAs -- adverse effects
- Molecular Mimicry
- Molecular Targeted Therapy
- Mutant Proteins -- drug effects
- Nerve Tissue Proteins -- drug effects
- Nuclear Proteins -- drug effects
- Phenotype
- Repressor Proteins -- drug effects
- Transduction, Genetic -- methods
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Publication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
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