Unesterified Cholesterol Accumulation in Late Endosomes/Lysosomes Causes Neurodegeneration and Is Prevented by Driving Cholesterol Export from This Compartment

Eitemau Tebyg

• Quantitative role of LAL, NPC2, and NPC1 in lysosomal cholesterol processing defined by genetic and pharmacological manipulations
  gan: Ramirez, Charina M., et al.
  Cyhoeddwyd: (2011)
• Reversal of defective lysosomal transport in NPC disease ameliorates liver dysfunction and neurodegeneration in the npc1(−/−) mouse
  gan: Liu, Benny, et al.
  Cyhoeddwyd: (2009)
• Multiple mechanisms limit the accumulation of unesterified cholesterol in the small intestine of mice deficient in both ACAT2 and ABCA1
  gan: Turley, Stephen D., et al.
  Cyhoeddwyd: (2010)
• Cyclodextrin overcomes the transport defect in nearly every organ of NPC1 mice leading to excretion of sequestered cholesterol as bile acid
  gan: Liu, Benny, et al.
  Cyhoeddwyd: (2010)
• Hepatic entrapment of esterified cholesterol drives continual expansion of whole body sterol pool in lysosomal acid lipase-deficient mice
  gan: Aqul, Amal, et al.
  Cyhoeddwyd: (2014)