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, Le CT, Nasser E, Li Y, Patel P, Zhuravka I, Lueptow LM, Tkachyova I, Xu S, Nissim I, Schulze A, Lipshutz GS.
Khoja S, et al. Among authors: zhuravka i.
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Mol Ther Methods Clin Dev. 2022.
PMID: 35505663
Free PMC article.