Objective
Leukodystrophies (LDs) are inherited rare neurodegenerative diseases of the white matter and its main component, the myelin, that are affecting predominantly children. Severity of the disease is related to the axonal dysfunction due to myelin deficiency or destruction. Despite the achievement of remarkable advances made in the past decade, there is no current curative therapy. The development of therapeutic approaches for myelin repair and neuroprotection constitutes the main objective of the LeukoTreat project. Indeed LDs constitute prototypic pathologies to tackle myelin formation/destruction issues as well as glial cells dysfunctions in neurodegeneration. The global aim is to promote the development of therapeutic strategies for the largest number of LD affected patients and further applications to more common white matter disorders and finally neurodegenerative diseases. For this purpose, the project will combine the expertise of (i) recognized European research teams in the field of White Matter diseases (COST Myelinet), (ii) high-technology SMEs, (iii) experts in medical ethics and (iv) LD patients and families associations. To develop efficient therapies, the LeukoTreat project is based on 5 complementary approaches consisting in: (i) collecting information on the epidemiology, the natural history, the genotype/phenotype correlation of LDs for at least 500 patients; (ii) validating/identifying biomarkers for therapeutic decisions/follow up to isolate new therapeutic targets; (iii) developing pharmacological strategies with the ultimate objective to launch at least 4 pharmacological clinical trials during 5 years following the project; (iv) developing innovative gene and cell therapies with the ultimate objective to launch at least 3 clinical trials during the next 5 years; (v) tackling ethical impacts of the proposed therapeutic challenges by integrating the participation of patients driven by a well-experienced research team strongly skilled in ethics
Fields of science
Call for proposal
FP7-HEALTH-2009-single-stage
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Funding Scheme
CP-FP - Small or medium-scale focused research projectCoordinator
63001 Clermont-Ferrand
France
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Participants (24)
00165 Roma
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16147 Genova
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53127 Bonn
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75654 Paris
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63360 Saint-Beauzire
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M13 9PL Manchester
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08908 L'Hospitalet De Llobregat
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80539 Munchen
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1090 Wien
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13288 MARSEILLE CEDEX 9
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WC1E 6BT LONDON
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20132 MILANO
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1105AZ Amsterdam
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75270 cedex 06 Paris
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75008 Paris
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54520 Laxou
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CB2 1TN Cambridge
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F-75205 / 13 Paris
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20133 Milano
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Participation ended
55131 Mainz
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20251 Hamburg
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72074 Tuebingen
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2052 Sydney
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20132 Milano
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