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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...
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. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2019
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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 |
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