Periodic Reporting for period 1 - ASTRO-EDITING (Targeting ASTROcytes with cutting-edge EDITING technologies to treat the Alexander disease)

Alexander disease (AxD) is an autosomal dominant neurodegenerative disorder caused by mutations in the gene encoding for the glial fibrillary acidic protein (GFAP), the major intermediate filament protein in astrocytes. In AxD patients, GFAP mutations are mainly clustered in...