Article

Effects of bromodomain and extraterminal domain protein inhibition in a mouse model of Niemann-Pick type C disease

2026-06-29

Abstract excerpt

Defects in lysosomal lipid handling provoke fatal disorders presenting neurovisceral symptoms with variable onset and life spans. A prime example is Niemann-Pick type C disease (NPCD), where export of cholesterol and other lipids from the endosomal-lysosomal system is impaired due to variants of either NPC intracellular cholesterol transporter 1 (NPC1) or NPC intracellular cholesterol transporter 2 (NPC2). Therape...

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Literature Corpus work: 91df59ea-6674-5c73-8892-4dec541c88e6
DOI: 10.64898/2026.06.24.734200

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