Corneal alterations during combined therapy with cyclodextrin/allopregnanolone and miglustat in a knock-out mouse model of NPC1 disease.

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...

Full description

Bibliographic Details
Main Authors: Marine Hovakimyan, Jana Petersen, Fabian Maass, Maria Reichard, Martin Witt, Jan Lukas, Oliver Stachs, Rudolf Guthoff, Arndt Rolfs, Andreas Wree
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2011-01-01
Series:PLoS ONE
Online Access:http://europepmc.org/articles/PMC3232193?pdf=render

Internet

http://europepmc.org/articles/PMC3232193?pdf=render

Similar Items