GENE THERAPY PREVENTS HEPATIC MITOCHONDRIAL DYSFUNCTION IN MURINE DEOXYGUANOSINE KINASE DEFICIENCY
Nandaki Keshavan, Miriam Greenwood, Helen Prunty, Juan F. Antinao Diaz, Riccardo Privolizzi, John Robert Counsell, Anna Karlsson, Neil James Sebire, Simon N. Waddington, Rajvinder Karda, Shamima Rahman
This study demonstrates that AAV9-mediated gene therapy prevents liver dysfunction in a mouse model of DGUOK deficiency.
Molecular Therapy — Methods & Clinical Development · Dec 13, 2024Peer-reviewedGenetics