AAV9-MCT8 delivery at juvenile stage ameliorates neurological and behavioral deficits in an AHDS mouse model
Xiao-Hui Liao, Pablo Avalos, Oksana Shelest, Raz Ofan, Michael Shilo, Catherine J. Bresee, Shibi B. Likhite, Jean‐Philippe Vit, Heike Heuer, Brian K. Kaspar, Kathrin Meyer, Alexandra M. Dumitrescu, Samuel Refetoff, Clive Niels Svendsen, Gad David Vatine
This study shows that juvenile AAV9-MCT8 gene therapy improves neurological and behavioral deficits in an AHDS mouse model by restoring TH levels and gene expression.
bioRxiv · Oct 31, 2021 · PreprintNeuroscience