Hepatic gene replacement restores energy metabolism and doubles the survival in mouse model of GRACILE syndrome, a neonatal mitochondrial disease
Rishi Banerjee, Janne Purhonen, Nasrin Sultana…·Jukka Kallijärvi
This study uses gene replacement in a mouse model of GRACILE syndrome to show improved energy metabolism and doubled survival through liver-specific correction of a mitochondrial complex III deficiency.