Objective
The EUCLYD consortium aims at developing a scientific network among outstanding communities of basic and clinical investigators in five European countries (Italy, Netherlands, UK, Sweden and Germany) to study various aspects of lysosomal storage diseases (LSD). LSDs are rare disorders (overall prevalence of 1/5000 to 1/8000), each due to a specific lysosomal enzyme deficiency, leading to intracellular storage of a variety of undegraded substrates (sphingolipids, glycosaminoglycans, glycoproteins and glycogen) in different tissues. Among the 40 to 50 LSDs presently known, EUCLYD will focus on Gaucher disease, Pompe disease, MPS VI and Multiple sulfatase deficiency, as prototypes of disorders with different stored materials in various organs and tissues outside the CNS. The issues to be investigated in the proposed project are: pathophysiology and mechanisms underlying the symptoms and leading to devastating clinical consequences; natural history; testing of novel therapeutic approaches. These issues will be addressed by patients studies and with mouse models recapitulating the phenotype of LSDs. Recent advances in the pathophysiology of LSDs point to the role of secondary processes triggered by substrate storage. A better understanding of these processes is crucial to identify new targets of therapy. Enzyme replacement and substrate reduction therapies were successfully introduced in the treatment of specific LSDs. However, pilot clinical trials should be supported by post-marketing studies to further assess the efficacy of these therapies and to develop standardized treatment protocols. To this purpose a precise knowledge of the natural history of LSDs is essential. Novel therapeutic strategies, such as enzyme enhancement and gene therapy are still under investigation in cell systems and in animal models. EUCLYD will provide further testing of these approaches in order to translate state of the art knowledge into clinical applications and reach patient's bedside.
Fields of science (EuroSciVoc)
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: The European Science Vocabulary.
- medical and health sciences medical biotechnology genetic engineering gene therapy
- humanities history and archaeology history
- medical and health sciences basic medicine physiology pathophysiology
- natural sciences biological sciences biochemistry biomolecules proteins enzymes
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Keywords
Project’s keywords as indicated by the project coordinator. Not to be confused with the EuroSciVoc taxonomy (Fields of science)
Project’s keywords as indicated by the project coordinator. Not to be confused with the EuroSciVoc taxonomy (Fields of science)
Programme(s)
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
Multi-annual funding programmes that define the EU’s priorities for research and innovation.
Topic(s)
Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Calls for proposals are divided into topics. A topic defines a specific subject or area for which applicants can submit proposals. The description of a topic comprises its specific scope and the expected impact of the funded project.
Call for proposal
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
FP7-HEALTH-2007-A
See other projects for this call
Funding Scheme
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
Funding scheme (or “Type of Action”) inside a programme with common features. It specifies: the scope of what is funded; the reimbursement rate; specific evaluation criteria to qualify for funding; and the use of simplified forms of costs like lump sums.
Coordinator
80138 Napoli
Italy
The total costs incurred by this organisation to participate in the project, including direct and indirect costs. This amount is a subset of the overall project budget.