Gene Therapy Rescues Brain Edema and Motor Function in a Mouse Model of Megalencephalic Leukoencephalopathy with Subcortical Cysts

To develop a gene therapy for megalencephalic leukoencephalopathy with subcortical cysts (MLC), scientists administered an adeno-associated viral vector capable of crossing the murine blood-brain barrier, delivering the human MLC1 cDNA under the control of a human astrocyte-specific promoter, to 10-month-old Mlc1-/- mice.