Gene therapy for guanidinoacetate methyltransferase deficiency restores cerebral and myocardial creatine while resolving behavioral abnormalities

by Eliav, A., Khoja, S., Lambert, J., Le, C.T, Li, Y., Lipshutz, G.S, Lueptow, L.M, Nasser, E., Nissim, I., Nitzahn, M., Patel, P., Schulze, A., Tamboline, M., Tkachyova, I., Xu, S., Zhang, Y., Zhuravka, I.
Published 2022