Combination therapy a potential strategy for treating Niemann Pick disease

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.
Source: ScienceDaily Headlines - Category: Science Source Type: news