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Linear Cyclodextrin Polymer Prodrugs as Novel Therapeutics for Niemann-Pick Type C1 Disorder

Niemann-Pick Type C1 disorder (NPC) is a rare lysosomal storage disease characterized by the accumulation of cholesterol in lysosomes. NPC has no FDA approved treatments yet, however 2-hydroxypropyl-β-cyclodextrin (HPβCD) has shown efficacy for treating the disease in both mouse and feline NPC model...

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Detalles Bibliográficos
Autores principales:	Kulkarni, Aditya, Caporali, Paola, Dolas, Atul, Johny, Soniya, Goyal, Sandeep, Dragotto, Jessica, Macone, Alberto, Jayaraman, Ramesh, Fiorenza, Maria Teresa
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group UK 2018
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6015065/ https://www.ncbi.nlm.nih.gov/pubmed/29934581 http://dx.doi.org/10.1038/s41598-018-27926-9

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