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

Abstract 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...

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Bibliographic Details
Published in:	BMC Medicine
Main Authors:	Mark L. Schultz, Maria V. Fawaz, Ruth D. Azaria, Todd C. Hollon, Elaine A. Liu, Thaddeus J. Kunkel, Troy A. Halseth, Kelsey L. Krus, Ran Ming, Emily E. Morin, Hayley S. McLoughlin, David D. Bushart, Henry L. Paulson, Vikram G. Shakkottai, Daniel A. Orringer, Anna S. Schwendeman, Andrew P. Lieberman
Format:	Article
Language:	English
Published:	BMC 2019-11-01
Subjects:	Niemann–Pick C NPC1 HDL SRS Niemann–Pick A
Online Access:	http://link.springer.com/article/10.1186/s12916-019-1423-5

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