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Genetic and Small Molecule Modifiers of Lysosomal Function

Objective

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.

Field of science

  • /natural sciences/biological sciences/cell biology
  • /medical and health sciences/health sciences/public and environmental health
  • /medical and health sciences/basic medicine/pathology
  • /medical and health sciences/basic medicine/medical genetics
  • /natural sciences/biological sciences/genetics and heredity/rna
  • /medical and health sciences/basic medicine/medicinal chemistry
  • /natural sciences/biological sciences/genetics and heredity
  • /natural sciences/biological sciences/biochemistry
  • /medical and health sciences/basic medicine/pharmacology and pharmacy
  • /natural sciences/biological sciences/biochemistry/biomolecules/proteins/proteomics

Call for proposal

H2020-MSCA-RISE-2016
See other projects for this call

Funding Scheme

MSCA-RISE - Marie Skłodowska-Curie Research and Innovation Staff Exchange (RISE)

Coordinator

INSTITUTO DE MEDICINA MOLECULAR JOAO LOBO ANTUNES
Address
Avenida Prof Egas Moniz
1649 028 Lisboa
Portugal
Activity type
Research Organisations
EU contribution
€ 366 300

Participants (9)

THE CHANCELLOR, MASTERS AND SCHOLARS OF THE UNIVERSITY OF OXFORD
United Kingdom
EU contribution
€ 198 000
Address
Wellington Square University Offices
OX1 2JD Oxford
Activity type
Higher or Secondary Education Establishments
THE UNIVERSITY OF SUSSEX
United Kingdom
EU contribution
€ 13 500
Address
Sussex House Falmer
BN1 9RH Brighton
Activity type
Higher or Secondary Education Establishments
WEIZMANN INSTITUTE OF SCIENCE
Israel
EU contribution
€ 18 450
Address
Herzl Street 234
7610001 Rehovot
Activity type
Higher or Secondary Education Establishments
FONDAZIONE TELETHON

Participation ended

Italy
EU contribution
€ 0
Address
Via Varese 16/B
00185 Roma
Activity type
Research Organisations
INSTITUTUL DE BIOCHIMIE
Romania
EU contribution
€ 130 500
Address
Splaiul Independentei No. 296
060031 Bucharest
Activity type
Research Organisations
LIPOTYPE
Germany
EU contribution
€ 0
Address
Tatzberg 47
01307 Dresden
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
GENOMED, DIAGNOSTICOS DE MEDICINA MOLECULAR SA
Portugal
EU contribution
€ 240 750
Address
Avenida Professor Egas Moniz
1600 190 Lisboa
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
CHRONOS BIOSYSTEMS SRL
Romania
EU contribution
€ 108 000
Address
Bulevardul Geniului, Nr 34, Parter, Camera 2
060117 Bucharest
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
INTRABIO LTD
United Kingdom
EU contribution
€ 27 000
Address
Summit House, 170 Finchley Road,
NW3 6BP London
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)

Partners (4)

PONTIFICIA UNIVERSIDAD CATOLICA DE CHILE
Chile
Address
Avenida Libertador Bernardo O'higgins 340
8331150 Santiago
Activity type
Higher or Secondary Education Establishments
UNIVERSIDAD DE BUENOS AIRES
Argentina
Address
Calle Viamonte 430
1053 Buenos Aires
Activity type
Higher or Secondary Education Establishments
THE CHILDREN'S HOSPITAL OF PHILADELPHIA NON PROFIT ORG
United States
Address
Civic Center Blvd
19104 Philadelphia
Activity type
Other
UNIVERSIDAD DEL DESARROLLO
Chile
Address
Av La Plaza 700
San Carlos De Apoquindo
Activity type
Higher or Secondary Education Establishments