טוען...
Antisense Oligonucleotide-Mediated Removal of the Polyglutamine Repeat in Spinocerebellar Ataxia Type 3 Mice
Spinocerebellar ataxia type 3 (SCA3) is a currently incurable neurodegenerative disorder caused by a CAG triplet expansion in exon 10 of the ATXN3 gene. The resultant expanded polyglutamine stretch in the mutant ataxin-3 protein causes a gain of toxic function, which eventually leads to neurodegener...
שמור ב:
| הוצא לאור ב: | Mol Ther Nucleic Acids |
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| Main Authors: | , , , |
| פורמט: | Artigo |
| שפה: | Inglês |
| יצא לאור: |
American Society of Gene & Cell Therapy
2017
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| נושאים: | |
| גישה מקוונת: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5504086/ https://ncbi.nlm.nih.gov/pubmed/28918024 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2017.06.019 |
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