GeneticsMolecular Therapy — Methods & Clinical DevelopmentPeer-reviewed
GENE THERAPY PREVENTS HEPATIC MITOCHONDRIAL DYSFUNCTION IN MURINE DEOXYGUANOSINE KINASE DEFICIENCY
Nandaki Keshavan, Miriam Greenwood, Helen Prunty…·Shamima Rahman
This study demonstrates that AAV9-mediated gene therapy prevents liver dysfunction in a mouse model of DGUOK deficiency.
6 citations