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Table 1 Effect of gemfibrozil on cells and on animals

From: Safety and potential efficacy of gemfibrozil as a supportive treatment for children with late infantile neuronal ceroid lipofuscinosis and other lipid storage disorders

  Treatment Effect Reference
Cells in vitro
 Mouse & human primary astrocytes gemfibrozi 25 μm ↑CLN2 mRNA ↑TPP1 protein 38, 39
 Mouse neurons from cortex, striatum & hippocampus gemfibrozil 25 μm ↑TPP1 protein 38
 Lymphoblastic cells from CLN3 patients gemfibrozil 25 μm ↑viability ↑autophagy recovery ↑autophagy genes 48
 Human IPS models CLN2 and CLN3 mutations gemfibrozil 25 µm No effect on TPP1 47
Animals
 Mouse oral gemfibrozil 7.5 mg/kg for 21 d ↑TTP1 in astrocytes, cortical neurons, & non neural cells of dentate gyrus & CAI of hippocampus 38
 Mouse KO LINCL oral gemfibrozil 7.5 mg/kg for 21 d ↑longevity, ↑motor retention, ↓apoptosis, ↑anti-inflammatory molecules 49
  1. d = days
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172