Influence of Npc1 genotype on the toxicity of hydroxypropyl-β-cyclodextrin, a potentially therapeutic agent, in Niemann–Pick Type C disease models()

Hydroxypropyl-β-cyclodextrin (HPBCD) is an attractive drug candidate against Niemann–Pick Type C (NPC) disease. However, the safety of HPBCD treatment for NPC patients remains to be elucidated. In this study, we examined the acute toxicity of HPBCD in Npc1-deficient mice. When treated with HPBCD (20...

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Veröffentlicht in:Mol Genet Metab Rep
Hauptverfasser: Tanaka, Yuta, Ishitsuka, Yoichi, Yamada, Yusei, Kondo, Yuki, Takeo, Toru, Nakagata, Naomi, Higashi, Taishi, Motoyama, Keiichi, Arima, Hidetoshi, Matsuo, Muneaki, Higaki, Katsumi, Ohno, Kousaku, Irie, Tetsumi
Format: Artigo
Sprache:Inglês
Veröffentlicht: Elsevier 2014
Schlagworte:
Research Paper
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5121301/
https://ncbi.nlm.nih.gov/pubmed/27896072
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2013.12.003
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