Gene therapy for guanidinoacetate methyltransferase deficiency restores cerebral and myocardial creatine while resolving behavioral abnormalities
胍基乙酸甲基转移酶缺乏症的基因治疗可恢复大脑和心肌肌酸，同时解决行为异常
期刊:Molecular Therapy-Methods & Clinical Development
影响因子:
doi:10.1016/j.omtm.2022.03.015
Suhail Khoja, Jenna Lambert, Matthew Nitzahn, Adam Eliav, YuChen Zhang, Mikayla Tamboline, Colleen T Le, Eram Nasser, Yunfeng Li, Puja Patel, Irina Zhuravka, Lindsay M Lueptow, Ilona Tkachyova, Shili Xu, Itzhak Nissim, Andreas Schulze, Gerald S Lipshutz
