LysoMod is a collaborative staff-exchange program between highly complementary and multidisciplinary academic and non-academic partners who have expertise in pharmacology, medicinal chemistry, cell biology, biochemistry, mouse and human genetics, transcriptomics, proteomics, and lipidomics. The LysoMod consortium also counts on a mentorship program that allows the exchange of young researchers among partner groups to prepare the next generation of researchers for careers either in the private or public health sectors.

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Considering the critical role that lysosomes play in cells, a better understanding of how lysosomes work will foster development of new treatments for people suffering from all types of lysosomal disfunction, from lysosomal storage diseases (LSDs) to age-related neurodegenerative disorders. New strategies to improve the quality of life of affected individuals may result from the LysoMod project.

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LysoMod’s specific objectives are:

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. to develop and further optimize existing therapies for LSDs;

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. to identify new targets for personalized therapies for LSDs;

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. to investigate the cross-talk between lysosomal function, signalling pathways, and gene expression regulation.

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Besides these specific objectives, the pioneering work of a member of the LysoMod consortium led to the development of a drug that is approved for clinical use. Therefore, the LysoMod also aims to:

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. to investigate the mechanisms of action of this and other drugs in lysosome  dysfunction and related disorders;

 

.to identify modifier genes involved in LSD pathology and test their potential as new targets for personalized therapeutic approaches;

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. to identify candidate RNAs that can be targeted to enhance lysosomal function.

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The non-academic partners of the LysoMod consortium have the role of facilitating knowledge transfer from research results obtained in academic labs into applications for developing diagnostic approaches and designing clinical trials.