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Lysosomal-specific Cholesterol Reduction by Biocleavable Polyrotaxanes for Ameliorating Niemann-Pick Type C Disease

Niemann-Pick type C (NPC) disease is an autosomal recessive lysosomal trafficking disorder, in which the cholesterols are abnormally accumulated in lysosomes. Recently, the β-cyclodextrin (CD) derivatives are revealed to show therapeutic effect for NPC disease through the removal of accumulated chol...

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Detalhes bibliográficos
Main Authors: Tamura, Atsushi, Yui, Nobuhiko
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2014
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3950578/
https://ncbi.nlm.nih.gov/pubmed/24619155
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/srep04356
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