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Therapeutic challenge in Leukodystrophies: Translational and ethical research towards clinical trials

Ziel

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

Wissenschaftliches Gebiet

Programm/Programme

Thema/Themen

Aufforderung zur Vorschlagseinreichung

FP7-HEALTH-2009-single-stage
Andere Projekte für diesen Aufruf anzeigen

Finanzierungsplan

CP-FP - Small or medium-scale focused research project

Koordinator

UNIVERSITE D'AUVERGNE CLERMONT-FERRAND 1
EU-Beitrag
€ 796 258,60
Adresse
BOULEVARD FRANCOIS MITTERAND 49
63001 Clermont-Ferrand
Frankreich

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Aktivitätstyp
Higher or Secondary Education Establishments
Kontakt Verwaltung
Isabelle Rhit
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Keine Daten

Beteiligte (24)

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Letzte Aktualisierung: 2 August 2019

Mein Booklet

Permalink: https://cordis.europa.eu/project/id/241622/de

European Union, 2024