Nanoparticles containing β‐cyclodextrin potentially useful for the treatment of Niemann‐Pick C

β‐Cyclodextrin (β‐CD) is being considered a promising therapy for Niemann‐Pick C (NPC) disease because of its ability to mobilise the entrapped cholesterol from lysosomes, however, a major limitation is its inability to cross the blood‐brain barrier (BBB) and address the central nervous system (CNS)...

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Veröffentlicht in:Journal of inherited metabolic disease 2020-05, Vol.43 (3), p.586-601
Hauptverfasser: Donida, Bruna, Raabe, Marco, Tauffner, Bárbara, Farias, Marcelo A., Machado, Andryele Z., Timm, Fernanda, Kessler, Rejane G., Hammerschmidt, Tatiane G., Reinhardt, Luiza S., Brito, Verônica B., Portugal, Rodrigo V., Bernardi, Andressa, Frozza, Rudimar, Moura, Dinara J., Giugliani, Roberto, Poletto, Fernanda, Vargas, Carmen R.
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Sprache:eng
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