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Antisense suppression of glial fibrillary acidic protein as a treatment for Alexander disease
OBJECTIVE: Alexander disease is a fatal leukodystrophy caused by autosomal dominant gain-of-function mutations in the gene for glial fibrillary acidic protein (GFAP), an intermediate filament protein primarily expressed in astrocytes of the central nervous system. A key feature of pathogenesis is ov...
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| Gepubliceerd in: | Ann Neurol |
|---|---|
| Hoofdauteurs: | , , , , , , , |
| Formaat: | Artigo |
| Taal: | Inglês |
| Gepubliceerd in: |
2018
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| Onderwerpen: | |
| Online toegang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5876100/ https://ncbi.nlm.nih.gov/pubmed/29226998 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ana.25118 |
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