NeuroscienceBrainPeer-reviewed
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…·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.
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