What is the Connection Between NPC1 and Neurotoxicity?
Neurotoxicity is a major concern in NPC1-related disorders. The central nervous system is particularly vulnerable to disruptions in cholesterol transport, given its high demand for cholesterol. Impaired NPC1 function can lead to neurotoxic effects due to the accumulation of cholesterol and other lipids in the brain. This not only contributes to the neurodegenerative symptoms seen in Niemann-Pick disease type C but may also influence the brain's response to neurotoxic agents.
