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Current Drug Metabolism


ISSN (Print): 1389-2002
ISSN (Online): 1875-5453

Review Article

Lysosomotropic Drugs: Pharmacological Tools to Study Lysosomal Function

Author(s): Sandra Pisonero-Vaquero and Diego Luis Medina*

Volume 18, Issue 12, 2017

Page: [1147 - 1158] Pages: 12

DOI: 10.2174/1389200218666170925125940

Price: $65


Background: Lysosomotropic molecules are taken up into lysosomes in vitro and in vivo. Many drugs approved for clinical medicine are lysosomotropic agents, characterized by promoting particular effects including cytoplasmic vacuolization, increase in number and size of lysosomes, inhibition of their enzymes and accumulation of undegraded material, leading mainly to phospholipidosis. Despite lysosomotropism has been extensively described and studied, the pathophysiological significance of this process is still not well understood.

Objetive: In this review, we focus on what is known about the effects of lysosomotropic drugs on specific lysosomal functions and their similarities with the phenotypic features of lysosomal storage disorders (LSDs).

Conclusion: Some effects of lysosomotropic drugs are very similar to pathologic features of human genetic diseases affecting lysosomal function, and therefore these drugs can be used as tools to understand the mechanisms underlying such patho-pathways as well as to create pharmacologically-induced models of LSDs.

Keywords: Lysosomotropism, LSDs, lysosome, lysosomal function, phospholipidosis, TFEB, autophagy.

Graphical Abstract

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