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Oligonucleotide therapy mitigates disease in Spinocerebellar Ataxia Type 3 mice
OBJECTIVE: Spinocerebellar ataxia type 3 (SCA3), also known as Machado-Joseph disease, is the most common dominantly inherited ataxia. Despite advances in understanding this CAG repeat/polyglutamine expansion disease, there are still no therapies to alter its progressive fatal course. Here we invest...
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| Publicado no: | Ann Neurol |
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| Main Authors: | , , , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2018
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6119475/ https://ncbi.nlm.nih.gov/pubmed/29908063 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ana.25264 |
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