Combination therapy a potential strategy for treating Niemann Pick disease

Thursday, May 15, 2014 - 19:02 in Biology & Nature

A potential dual-pronged approach to treating Niemann-Pick type C (NPC) disease, a rare but devastating genetic disorder, has been identified by researchers. By studying nerve and liver cells grown from NPC patient-derived induced pluripotent stem cells (iPSCs), the scientists determined that although cholesterol does accumulate abnormally in the cells of NPC patients, a more significant problem may be defective autophagy -— a basic cellular function that degrades and recycles unneeded or faulty molecules, components, or organelles in a cell.

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