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...
Topics
- Alexander Disease
- Animals
- Biomarkers
- Brain Chemistry
- Gene Expression Regulation
- Glial Fibrillary Acidic Protein
- Hippocampus
- Humans
- Injections, Intraventricular
- Mice
- Mice, Inbred C57BL
- Mutation
- Neurogenesis
- Oligonucleotides, Antisense