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Content archived on 2024-05-29

Embryonic stem cells for therapy and exploration of mechanisms in Huntington disease

Objective

Embryonic stem cell (ES) lines have potential interest for exploring mechanisms and therapeutics of genetically-determined diseases, due to their fundamental attributes, unlimited expansion and pluripotency: isogenic cells of any phenotype can be produced in any requested amount. STEM-HD aims at validating this potential for Huntington's disease (HD), on the basis of the analysis of ES cell lines expressing the mutant HD gene. These will be obtained either following derivation after pre-implantation genetic diagnosis from an embryo expressing the mutant hunting tin gene (existing cell line) or through engineering of existing native human and mouse ES cell lines.

The consortium will:
- establish protocols to enrich ES cell progeny in cell phenotypes of interest, in particular striatal GABA neurons.
- design and implement infrastructures for mass cell production and long-term cultures of ES cells either undifferentiated or following guided differentiation.
- identify, using large-scale "resource-driven" approaches, HD biomarkers in mutant HD-expressing ES cells progeny appearing as alteration in the expression of genes (transcriptome). Mutant-association of these molecular markers will be validated using patients' samples and assays developed.
- explore the mechanisms of the diseases in the mutant HD-expressing ES cells progeny using functional genomics (RNAi and gene over-expression) and "hypothesis-driven" approaches.
- perform high content drug screening on HD gene-carrying ES cell progeny using validated biomarkers as signals and available compounds libraries.

Fields of science (EuroSciVoc)

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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.

Call for proposal

Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.

FP6-2005-LIFESCIHEALTH-6
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Funding Scheme

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STREP - Specific Targeted Research Project

Coordinator

INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE
EU contribution
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Total cost

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Participants (7)

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