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Synthetic high-density lipoprotein nanoparticles for the treatment of Niemann–Pick diseases

BACKGROUND: Niemann–Pick disease type C is a fatal and progressive neurodegenerative disorder characterized by the accumulation of unesterified cholesterol in late endosomes and lysosomes. We sought to develop new therapeutics for this disorder by harnessing the body’s endogenous cholesterol scaveng...

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Detalles Bibliográficos
Autores principales: Schultz, Mark L., Fawaz, Maria V., Azaria, Ruth D., Hollon, Todd C., Liu, Elaine A., Kunkel, Thaddeus J., Halseth, Troy A., Krus, Kelsey L., Ming, Ran, Morin, Emily E., McLoughlin, Hayley S., Bushart, David D., Paulson, Henry L., Shakkottai, Vikram G., Orringer, Daniel A., Schwendeman, Anna S., Lieberman, Andrew P.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6849328/
https://www.ncbi.nlm.nih.gov/pubmed/31711490
http://dx.doi.org/10.1186/s12916-019-1423-5