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DURABLE AND DOSE-DEPENDENT CORRECTION OF THE BIOCHEMICAL PHENOTYPE IN A MOUSE MODEL OF GUANIDINOACETATE METHYLTRANSFERASE (GAMT) CREATINE DEFICIENCY FOLLOWING TREATMENT WITH SCAAV9.HGAMT

Robyn T. Binsfeld, Troy B. Webster,Ilona Tkachyova, Emahnee Cover, Tesla Peretti,Brianna M. Quinville,Melissa Mitchell,Andreas Schulze,Jagdeep S. Walia

Cytotherapy（2024）

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关键词
Creatine Deficiency,AAV9,Inborn Errors of Metabolism
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