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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Veröffentlicht in:Ann Neurol
Hauptverfasser: Hagemann, Tracy L., Powers, Berit, Mazur, Curt, Kim, Aneeza, Wheeler, Steven, Hung, Gene, Swayze, Eric, Messing, Albee
Format: Artigo
Sprache:Inglês
Veröffentlicht: 2018
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Article
Online Zugang: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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