In megalencephalic leukoencephalopathy, in which astrocytes are particularly affected, treatment of Mlc1 knockout mice through injection of rAAV.rh10 GFAP promoter-driven MLC1 vectors in the cerebrospinal fluid resulted in MLC1 expression in the cerebellum and improved tissue pathology by reinstating the proper localization of the adhesion molecule GlialCAM and the chloride channel ClC-2 within Bergmann glia. Here, MLC1 is linked to leukoencephalopathy, megalencephalic.