Gene therapy targeting the blood-brain barrier improves neurological symptoms in a model of genetic MCT8 deficiency.

Sundaram SM, Arrulo Pereira A, Müller-Fielitz H, Köpke H, De Angelis M, Müller TD, Heuer H, Körbelin J, Krohn M, Mittag J, Nogueiras R, Prevot V, Schwaninger M.