EFFECTS OF N-ACETYL-LEUCINE AND ITS ENANTIOMERS IN NIEMANN-PICK DISEASE TYPE C CELLS

N-Acetyl-DL-Leucine and N-Acetyl-L-Leucine reduced the relative lysosomal volume in NPC1-/- Chinese Hamster Ovary cells in a dose-dependent manner. N-Acetyl-L-Leucine was most effective at reducing relative lysosomal volumes in fibroblasts derived from NPC patients with severe disease (***p <0.001), followed by N-Acetyl-DL-Leucine (**p <0.01). Treatment with N-Acetyl-D-Leucine did not achieve statistical significance.

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