Gene delivery of AGAT and GAMT boosts creatine levels in creatine transporter deficiency patient fibroblasts.

After three days, for the first CT1 mutant line ( SLC6A8 Δex10-11/y ), we observed a 3.5-fold increase in intracellular creatine although this increase just failed to reach statistical significance ( Fig 4C , 4.2 vs 1.2 nmoles/mg protein relative to empty vector controls, P = 0.061, two-sided t-test).

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