In a different approach, a single intrathecal injection of a GFAP mRNA targeted ASO with enhanced nuclease resistance, binding affinity and lower toxicity has achieved remarkable efficacy providing elimination of GFAP throughout the CNS, reversal of Rosenthal fibers and rescue of hippocampal neurogenesis in an AxD mouse model (Hagemann et al., 2018). The gene discussed is GFAP; the disease is Alexander disease.