Gene therapy targeting the blood-brain barrier improves neurological symptoms in a model of genetic MCT8 deficiency
Sivaraj Mohana Sundaram, Adriana Arrulo Pereira, Helge Müller‐Fielitz, Hannes Köpke, Meri De Angelis, Timo D. Müller, Heike Heuer, Jakob Körbelin, Markus Krohn, Jens Mittag, Rubén Nogueiras, Vincent Prévot, Markus Schwaninger
This study demonstrates that gene therapy targeting brain endothelial cells improves neurological symptoms in a mouse model of MCT8 deficiency by increasing brain T3 levels and motor function.
Brain · Aug 5, 2022Peer-reviewedNeuroscience