Hydroxypropyl-beta and -gamma cyclodextrins rescue cholesterol accumulation in Niemann–Pick C1 mutant cell via lysosome-associated membrane protein 1
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چکیده
منابع مشابه
Correction: Characterization of hydroxypropyl-beta-cyclodextrins used in the treatment of Niemann-Pick Disease type C1
[This corrects the article DOI: 10.1371/journal.pone.0175478.].
متن کاملCharacterization of hydroxypropyl-beta-cyclodextrins used in the treatment of Niemann-Pick Disease type C1
2-Hydroxypropyl-beta-cyclodextrin (HPβCD) has gained recent attention as a potential therapeutic intervention in the treatment of the rare autosomal-recessive, neurodegenerative lysosomal storage disorder Niemann-Pick Disease Type C1 (NPC1). Notably, HPβCD formulations are not comprised of a single molecular species, but instead are complex mixtures of species with differing degrees of hydroxyp...
متن کاملGlycosylation regulates the expression of Lysosome Associated Membrane Protein-1 (LAMP1) on the cell surface
Lysosome Associated Membrane protein-1 (LAMP1) which lines the lysosomes, is often found to appear on the surface of several cells involved in migratory and/or invasive functions including metastatic tumor cells. However, the mechanism of its surface translocation in these cells is still poorly understood. Glycosylation, one of the major post-translational modifications of membrane proteins, re...
متن کامل2-Hydroxypropyl-β-cyclodextrins and the Blood-Brain Barrier: Considerations for Niemann-Pick Disease Type C1
The rare, chronic, autosomal-recessive lysosomal storage disease Niemann-Pick disease type C1 (NPC1) is characterized by progressively debilitating and ultimately fatal neurological manifestations. There is an urgent need for disease-modifying therapies that address NPC1 neurological pathophysiology, and passage through the blood-brain barrier represents an important consideration for novel NPC...
متن کاملCholesterol Transport through Lysosome-Peroxisome Membrane Contacts
Cholesterol is dynamically transported among organelles, which is essential for multiple cellular functions. However, the mechanism underlying intracellular cholesterol transport has remained largely unknown. We established an amphotericin B-based assay enabling a genome-wide shRNA screen for delayed LDL-cholesterol transport and identified 341 hits with particular enrichment of peroxisome gene...
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ژورنال
عنوان ژورنال: Cell Death & Disease
سال: 2018
ISSN: 2041-4889
DOI: 10.1038/s41419-018-1056-1