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Alterations in endo-lysosomal function induce similar hepatic lipid profiles in rodent models of drug-induced phospholipidosis and Sandhoff disease

Accepted version
Peer-reviewed

Type

Article

Change log

Authors

Lecommandeur, E 
Baker, D 
Cox, TM 
Nicholls, AW 
Griffin, JL 

Abstract

Drug-induced phospholipidosis (DIPL) is characterized by an increase in the phospholipid content of the cell and the accumulation of drugs and lipids inside the lysosomes of affected tissues, including in the liver. Although of uncertain pathological significance for patients, the condition remains a major impediment for the clinical development of new drugs. Human Sandhoff disease (SD) is caused by inherited defects of the β subunit of lysosomal β-hexosaminidases (Hex) A and B, leading to a large array of symptoms, including neurodegeneration and ultimately death by the age of 4 in its most common form. The substrates of Hex A and B, gangliosides GM2 and GA2, accumulate inside the lysosomes of the CNS and in peripheral organs. Given that both DIPL and SD are associated with lysosomes and lipid metabolism in general, we measured the hepatic lipid profiles in rodent models of these two conditions using untargeted LC/MS to examine potential commonalities. Both model systems shared a number of perturbed lipid pathways, notably those involving metabolism of cholesteryl esters, lysophosphatidylcholines, bis(monoacylglycero)phosphates, and ceramides. We report here profound alterations in lipid metabolism in the SD liver. In addition, DIPL induced a wide range of lipid changes not previously observed in the liver, highlighting similarities with those detected in the model of SD and raising concerns that these lipid changes may be associated with underlying pathology associated with lysosomal storage disorders.

Description

Keywords

ceramides, lipidomics, lysophospholipid, lysosome, mass spectrometry, storage diseases, toxicology

Journal Title

Journal of Lipid Research

Conference Name

Journal ISSN

0022-2275
1539-7262

Volume Title

58

Publisher

American Society for Biochemistry and Molecular Biology
Sponsorship
MRC (unknown)
Sparks: The Children's Medical Research Charity (09CAM01)
Wellcome Trust (093148/Z/10/Z)
National Institute of Diabetes and Digestive and Kidney Diseases (R21DK070288)
Medical Research Council (MR/P011705/1)
Medical Research Council (MR/P01836X/1)
Medical Research Council (G0801841)
Medical Research Council (G0801841/1)
This work was funded by a Medical Research Council Integrative Toxicology Training Partnership grant with financial support from GlaxoSmithKline. The work on Sandhoff mice was supported by SPARKS, The Children’s Medical Research Charity. JLG’s laboratory is supported by the Wellcome Trust (Equipment grant 093,148/Z/10/Z)) and the Medical Research Council (G0801841 & UD99999906).