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Genetic and Small Molecule Modifiers of Lysosomal Function

Funding Agency European Commission
Funding Programme Horizon 2020
Call/Topic H2020-MSCA-RISE-2016
Project reference 734825
Start date 2017.03.01
Duration 36 months
Total investment EUR 1 156 500(EU contribution: EUR 1 102 500)
Project Coordinator Maria Carmo-Fonseca
Project Beneficiaries

Instituto de Medicina Molecular João Lobo Antunes (iMM), Portugal
University of Oxford, United Kingdom
University of Sussex, United Kingdom
Weizmann Institute of Science, Israel
Romanian Academy - Institute of Biochemistry, Romania
Lipotype, Germany
GenoMed, Portugal
Chronos Biosystems, Romania
IntraBio, United Kingdom

Project Partners

Pontifícia Universidad Catolica de Chile, Chile
Universidad del Desarrollo, Chile
Universidad de Buenos Aires, Argentina
The Children’s Hospital of Philadelphia, USA


This project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 734825.

  • Overview

    LysoMod will innovate in the area of personalized medicine for disorders linked to lysosomal dysfunction. This will be achieved by implementing a collaborative staff-exchange program between highly complementary and multidisciplinary academic and non-academic partners with expertise in pharmacology, medicinal chemistry, cell biology, biochemistry, mouse and human genetics, transcriptomics, proteomics and lipidomics. Based on the critical role that lysosomes play in cells, a better understanding of lysosomal function will have a major impact on human health, fostering the development of new strategies to improve quality of life for people affected by a variety of diseases, ranging from lysosomal storage diseases (LSDs) to age-related neurodegenerative disorders. LysoMod’s specific objectives are: 1) to develop and further optimize existing therapies for LSDs; 2) to identify new targets for personalized therapies for LSDs; and 3) to investigate the cross-talk between lysosomal function, signalling pathways and gene expression regulation. The pioneer work of a participant in the consortium led to the development of a drug that is approved for clinical use. LysoMod will i) investigate the mechanisms of action of this and other drugs in lysosome-related disorder; ii) identify modifier genes involved in LSD pathology and test their potential as new targets for personalized therapeutic approaches; iii) identify candidate RNAs that can be targeted to enhance lysosomal function. The companies in the consortium will ensure a rapid transfer of new knowledge into applications for diagnostics and clinical trials. Prioritising lysosomal dysfunction as a highly relevant biomedical problem, the LysoMod consortium will implement a mentored staff-exchange program to provide young researchers with high-level training in innovative approaches for exploring biological systems, preparing the next generation of researchers for careers either in the private or public health sectors.

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