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Antisense oligonucleotide therapy for spinocerebellar ataxia type 2
Adult human neurodegenerative diseases have no disease-modifying treatments. We used spinocerebellar ataxia type 2 (SCA2), an autosomal dominant polyglutamine disease1, as a model to test RNA-targeted therapies(2) in two SCA2 mouse models. Both models recreate progressive adult-onset dysfunction and...
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| Publicado no: | Nature |
|---|---|
| Main Authors: | , , , , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2017
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6625650/ https://ncbi.nlm.nih.gov/pubmed/28405024 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/nature22044 |
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