Synergistic effect of deoxynucleosides and AAV gene therapy for thymidine kinase 2 deficiency
Carlos Lopez Gomez, Maria J. Sanchez‐Quintero, Eung Jeon Lee, Giulio Kleiner, Jun Xie, Hasan Orhan Akman, Guangping Gao, Michio Hirano
This study shows that combining AAV gene therapy with deoxynucleoside supplementation improves survival and mitochondrial function in Tk2-deficient mice.
bioRxiv · Oct 8, 2020 · PreprintGenetics