Article
Antisense suppression of glial fibrillary acidic protein as a treatment for Alexander disease.
Annals of neurology - 1 Jan 2018
Hagemann Tracy L, Powers Berit, Mazur Curt, Kim Aneeza, Wheeler Steven, Hung Gene, Swayze Eric, Messing Albee
Abstract excerpt
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 overexpression and accumulation of GFAP, with formation of characteristic cytoplasmic aggregates known as Rosenthal...
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