(Medical Xpress)—Whitehead Institute researchers have determined that the lipid storage disorder Niemann-Pick type C1 (NPC1) disease is caused not only by defects in cholesterol processing but also in autophagy—a key cellular degradation pathway that malfunctions in many neurodegenerative diseases. Targeting both the cholesterol accumulation and stalled autophagy found in NPC1 with combination therapy could represent a viable treatment strategy.
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