NeurosciencebioRxivPreprint
AAV9-MCT8 delivery at juvenile stage ameliorates neurological and behavioral deficits in an AHDS mouse model
Xiao-Hui Liao, Pablo Avalos, Oksana Shelest…·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.
DOI 10.1101/2021.10.31.466634