Kazuyuki Kitatani

A Colletotrichum orbiculare-based fungal model recapitulates conserved cellular defects of Niemann–Pick type C disease and enables therapeutic evaluation

Abstract Niemann–Pick type C disease is a fatal lysosomal storage disorder caused by mutations in NPC1 or NPC2 , leading to lipid accumulation and progressive neurodegeneration. Current mammalian models recapitulate major pathological features but are limited by long disease cour...