Corneal Alterations during Combined Therapy with Cyclodextrin/Allopregnanolone and Miglustat in a Knock-Out Mouse Model of NPC1 Disease

BACKGROUND: Niemann Pick disease type C1 is a neurodegenerative disease caused by mutations in the NPC1 gene, which result in accumulation of unesterified cholesterol and glycosphingolipids in the endosomal-lysosomal system as well as limiting membranes. We have previously shown the corneal involvem...

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Autors principals: Hovakimyan, Marine, Petersen, Jana, Maass, Fabian, Reichard, Maria, Witt, Martin, Lukas, Jan, Stachs, Oliver, Guthoff, Rudolf, Rolfs, Arndt, Wree, Andreas
Format: Artigo
Idioma:Inglês
Publicat: Public Library of Science 2011
Matèries:
Research Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3232193/
https://ncbi.nlm.nih.gov/pubmed/22163015
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0028418
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