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环糊精接枝治疗诊断纳米颗粒可改善血脑屏障模型穿越。

Grafting of Cyclodextrin to Theranostic Nanoparticles Improves Blood-Brain Barrier Model Crossing.

机构信息

Department of Bionanosciences, Institute of Biologically Inspired Materials, University of Natural Resources and Life Sciences (BOKU), 1190 Vienna, Austria.

Dipartimento di Scienze Chimiche, Università degli Studi di Catania, 95125 Catania, Italy.

出版信息

Biomolecules. 2023 Mar 22;13(3):573. doi: 10.3390/biom13030573.

DOI:10.3390/biom13030573
PMID:36979508
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10046162/
Abstract

Core-shell superparamagnetic iron oxide nanoparticles hold great promise as a theranostic platform in biological systems. Herein, we report the biological effect of multifunctional cyclodextrin-appended SPIONs (CySPION) in mutant Npc1-deficient CHO cells compared to their wild type counterparts. CySPIONs show negligible cytotoxicity while they are strongly endocytosed and localized in the lysosomal compartment. Through their bespoke pH-sensitive chemistry, these nanoparticles release appended monomeric cyclodextrins to mobilize over-accumulated cholesterol and eject it outside the cells. CySPIONs show a high rate of transport across blood-brain barrier models, indicating their promise as a therapeutic approach for cholesterol-impaired diseases affecting the brain.

摘要

核壳型超顺磁性氧化铁纳米粒子作为一种治疗诊断一体化平台在生物系统中具有广阔的应用前景。在此,我们报告了多功能环糊精修饰的超顺磁性氧化铁纳米粒子(CySPION)在突变 NPC1 缺陷型 CHO 细胞中的生物学效应,与野生型细胞相比。CySPION 表现出可以忽略不计的细胞毒性,同时它们被强烈内吞并定位于溶酶体区室中。通过其定制的 pH 敏感化学性质,这些纳米粒子释放出附着的单体环糊精,以动员过度积累的胆固醇并将其排出细胞外。CySPION 显示出穿过血脑屏障模型的高转运率,这表明它们有希望成为治疗影响大脑的胆固醇相关疾病的一种方法。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3cce/10046162/fec89aaa4fe5/biomolecules-13-00573-g006.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3cce/10046162/5fc5bfd2a037/biomolecules-13-00573-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3cce/10046162/c93e2869abc9/biomolecules-13-00573-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3cce/10046162/5eebe5985399/biomolecules-13-00573-g003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3cce/10046162/03bbba68d01c/biomolecules-13-00573-g004.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3cce/10046162/7605287b52cb/biomolecules-13-00573-g005.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3cce/10046162/fec89aaa4fe5/biomolecules-13-00573-g006.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3cce/10046162/5fc5bfd2a037/biomolecules-13-00573-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3cce/10046162/c93e2869abc9/biomolecules-13-00573-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3cce/10046162/5eebe5985399/biomolecules-13-00573-g003.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3cce/10046162/03bbba68d01c/biomolecules-13-00573-g004.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3cce/10046162/7605287b52cb/biomolecules-13-00573-g005.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3cce/10046162/fec89aaa4fe5/biomolecules-13-00573-g006.jpg

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Long-term survival outcomes of patients with Niemann-Pick disease type C receiving miglustat treatment: A large retrospective observational study.
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