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

Autor: Binsfeld, R.T., Webster, T., Tkachyova, I., Cover, E., Peretti, T., Quinville, B., Mitchell, M., Schulze, A., Walia, J.S.
Zdroj: In Cytotherapy June 2024 26(6) Supplement:S27-S27
Databáze: ScienceDirect
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