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In Vitro and In Vivo Evaluation of 6-O-α-Maltosyl-β-Cyclodextrin as a Potential Therapeutic Agent Against Niemann-Pick Disease Type C

Niemann-Pick disease Type C (NPC) is a rare lysosomal storage disease characterized by the dysfunction of intracellular cholesterol trafficking with progressive neurodegeneration and hepatomegaly. We evaluated the potential of 6-O-α-maltosyl-β-cyclodextrin (G2-β-CD) as a drug candidate against NPC....

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Pubblicato in:Int J Mol Sci
Autori principali: Yasmin, Nushrat, Ishitsuka, Yoichi, Fukaura, Madoka, Yamada, Yusei, Nakahara, Shuichi, Ishii, Akira, Kondo, Yuki, Takeo, Toru, Nakagata, Naomi, Motoyama, Keiichi, Higashi, Taishi, Okada, Yasuyo, Nishikawa, Junichi, Ichikawa, Atsushi, Iohara, Daisuke, Hirayama, Fumitoshi, Higaki, Katsumi, Ohno, Kousaku, Matsuo, Muneaki, Irie, Tetsumi
Natura: Artigo
Lingua:Inglês
Pubblicazione: MDPI 2019
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC6429330/
https://ncbi.nlm.nih.gov/pubmed/30845767
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms20051152
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