Pluronic based β-cyclodextrin polyrotaxanes for treatment of Niemann-Pick Type C disease

Niemann-Pick Type C disease (NPC) is a rare metabolic disorder characterized by disruption of normal cholesterol trafficking within the cells of the body. There are no FDA approved treatments available for NPC patients. Recently, the cycloheptaglucoside 2-hydroxypropyl-β-cyclodextrin (HP-β-CD) has s...

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Veröffentlicht in:Sci Rep
Hauptverfasser: Collins, Christopher J., Loren, Bradley P., Alam, Md Suhail, Mondjinou, Yawo, Skulsky, Joseph L., Chaplain, Cheyenne R., Haldar, Kasturi, Thompson, David H.
Format: Artigo
Sprache:Inglês
Veröffentlicht: Nature Publishing Group 2017
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Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5408228/
https://ncbi.nlm.nih.gov/pubmed/28452365
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/srep46737
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