Gene therapy for guanidinoacetate methyltransferase deficiency restores cerebral and myocardial creatine while resolving behavioral abnormalities
Khoja, S., Lambert, J., Nitzahn, M., Eliav, A., Zhang, Y., Tamboline, M., . . . Lipshutz, G. S. (2022). Gene therapy for guanidinoacetate methyltransferase deficiency restores cerebral and myocardial creatine while resolving behavioral abnormalities. Molecular Therapy – Methods & Clinical Development, 25, 278-296. doi:10.1016/j.omtm.2022.03.015