Gene therapy decreases seizures in a model of Incontinentia pigmenti.
Godwin K DogbeviaKathrin TöllnerJakob KörbelinSonja BröerDirk A RidderHanna GrasshoffClaudia BrandtJan WenzelBeate K StraubMartin TrepelWolfgang LöscherMarkus SchwaningerPublished in: Annals of neurology (2017)
The data show that the BBB is a target of antiepileptic treatment and, more specifically, provide evidence for the therapeutic benefit of a brain endothelial-targeted gene therapy in IP. Ann Neurol 2017;82:93-104.