Objective
Huntington's is a devastating neurodegenerative disease with many unmet patient needs. There are no known ways of slowing or preventing the neuro-degeneration, and clinical trials in man are hampered by the slow disease progression and the absence of suitable biomarkers of short-term progression. The genetics of HD is characterized, and involves the expansion of a polyglutamine tract at the amino-terminus of the Huntington gene (HTT). However, the translation of this knowledge into therapeutic and diagnostic approaches is hampered by the scarce knowledge of HTT biology, the paucity of information on how cellular signalling pathways interact with the HD mutation, and the lack of systematic and modern approaches aimed at identifying useful bio-predictors of disease progression in individuals diagnosed with HD.
The proposal addresses key areas of HD patient need, namely the discovery and development of therapeutically meaningful novel targets and biomarkers. To achieve these aims, this consortium representing complementary and specific know-how and expertise which will be integrated and further developed in the course of the project. High throughput-RNAi, focusing on genes encoding pharmacologically tractable proteins rather than on a whole-genome approach, will be employed on a novel and robust HD cellular disease model to identify proteins whose inhibition of expression is protective against the HD mutation. Following a stringent target validation approach, two such validated targets will be selected for assay development and primary screening activities, to identify druggable compounds active on the target and efficacious against the HD mutation in cellular disease models. In parallel, the biomaterial repository made available to the consortium from a disease-specialist academic partner will be investigated through state-of-the-art metabonomics approaches to identify biomarkers predictive of disease onset.
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.
- natural sciences biological sciences neurobiology
- natural sciences biological sciences biochemistry biomolecules proteins
- natural sciences biological sciences genetics mutation
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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.
FP6-2005-LIFESCIHEALTH-7
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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
SIENA
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.