Organ Weights in <i>NPC1</i> Mutant Mice Partly Normalized by Various Pharmacological Treatment Approaches

Organ Weights in <i>NPC1</i> Mutant Mice Partly Normalized by Various Pharmacological Treatment Approaches

Niemann-Pick Type C1 (NPC1, MIM 257220) is a rare, progressive, lethal, inherited autosomal-recessive endolysosomal storage disease caused by mutations in the <i>NPC1</i> leading to intracellular lipid storage. We analyzed mostly not jet known alterations of the weights of 14 different o...

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Bibliographic Details
Main Authors: Veronica Antipova, Lisa-Marie Steinhoff, Carsten Holzmann, Arndt Rolfs, Carlos Junior Hempel, Martin Witt, Andreas Wree
Format: Article
Language:English
Published: MDPI AG 2022-12-01
Series:International Journal of Molecular Sciences
Subjects:
NPC1
lipid storage disorder
treatment effect
miglustat
2-hydroxypropyl-β-cyclodextrin
allopregnanolone
Online Access:https://www.mdpi.com/1422-0067/24/1/573

Internet

https://www.mdpi.com/1422-0067/24/1/573

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