Advances in research on potential therapeutic approaches for Niemann-Pick C1 disease

Niemann-Pick disease type C1 (NP-C1) is a rare and devastating recessive inherited lysosomal lipid and cholesterol storage disorder caused by mutations in the NPC1 or NPC2 gene. These two proteins bind to cholesterol and cooperate in endosomal cholesterol transport. Characteristic clinical manifesta...

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Main Authors: Caifeng Zhang (Author), Keke Su (Author), Xu Jiang (Author), Yuping Tian (Author), Ke Li (Author)
Format: Book
Published: Frontiers Media S.A., 2024-08-01T00:00:00Z.
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3rd Floor Main Library

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