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, Oliver Ros, Anni I. Nieminen, Christa Kietz, Vineta Fellman, 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.
Molecular Therapy · Apr 25, 2026Peer-reviewedPhysiology