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RNA Interference-Based Therapy for Spinocerebellar Ataxia Type 7 Retinal Degeneration
Spinocerebellar ataxia type 7 (SCA7) is an autosomal dominant neurodegenerative disease characterized by loss of motor coordination and retinal degeneration with no current therapies in the clinic. The causative mutation is an expanded CAG repeat in the ataxin-7 gene whose mutant protein product cau...
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| Главные авторы: | , , , , , , , |
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| Формат: | Artigo |
| Язык: | Inglês |
| Опубликовано: |
Public Library of Science
2014
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| Предметы: | |
| Online-ссылка: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3997397/ https://ncbi.nlm.nih.gov/pubmed/24759684 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0095362 |
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