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Structure-function relationships of cholesterol mobilization from the endo-lysosome compartment of NPC1-deficient human cells by β-CD polyrotaxanes

Niemann-Pick Type C is a rare metabolic disorder characterized by the cellular accumulation of cholesterol within endosomal and lysosomal compartments. 2-Hydroxypropyl-β-cyclodextrin (HP-β-CD) containing polyrotaxanes represent an attractive approach for treating this disease due to their ability to...

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Detalles Bibliográficos
Autores principales: Samaddar, Shayak, Bose, Debosreeta, Loren, Bradley P., Skulsky, Joseph L., Ilnytska, Olga, Struzik, Zachary J., Storch, Judith, Thompson, David H.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9803220/
https://www.ncbi.nlm.nih.gov/pubmed/36584173
http://dx.doi.org/10.1371/journal.pone.0268613