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Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse Models
The most common dominantly inherited ataxia, spinocerebellar ataxia type 3 (SCA3), is an incurable neurodegenerative disorder caused by a CAG repeat expansion in the ATXN3 gene that encodes an abnormally long polyglutamine tract in the disease protein, ATXN3. Mice lacking ATXN3 are phenotypically no...
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| Publicado no: | Mol Ther Nucleic Acids |
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| Main Authors: | , , , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
American Society of Gene & Cell Therapy
2017
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5415970/ https://ncbi.nlm.nih.gov/pubmed/28624196 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2017.04.005 |
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