Objective
"Familial hypercholesterolemia type IIa (FH) is a life-threatening disease caused by mutations on the Low Density Lipoprotein Receptor (LDLR). While drug treatments reduce the level of circulating cholesterol in heterozygote patients (1:500) with variable efficiency, homozygote patients (1:106) can only be treated by bimonthly apheresis, an invasive procedure. Hepatocytes are the only cells capable to internalize and degrade cholesterol.
We previously shown that we were able to generated iPS from normal human fibroblasts and differentiated them into hepatocyte-like cells.
Our aim is to develop preclinical tools and models to study human monogenic liver metabolic diseases and evaluate the ex vivo gene/cell therapy approaches.
Our study will be focused on patient specific model for FH. In addition to reproducing the pathology in vitro with hepatocyte-like cells-derived from heterozygote and homozygote FH patients, this model will allow us to study the impact of LDLR mutations on cholesterol homeostasis in a human context. At a more long-term, FH-derived hepatocytes should represent alternative tools for drug screening in an actual current context in which there are no cellular models displaying features of hepatocytes. They will enable testing the efficacy of new drugs on cholesterol pathway and their effects on the specific disease-related hepatocyte phenotype (cell differentiation and proliferation). Finally, this model will provide a tool to evaluate autologous gene/cell therapy with iPS-derived hepatocyte-like cells."
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: https://op.europa.eu/en/web/eu-vocabularies/euroscivoc.
CORDIS classifies projects with EuroSciVoc, a multilingual taxonomy of fields of science, through a semi-automatic process based on NLP techniques. See: https://op.europa.eu/en/web/eu-vocabularies/euroscivoc.
- medical and health sciences medical biotechnology cells technologies stem cells
- natural sciences biological sciences genetics mutation
- medical and health sciences basic medicine pathology
- medical and health sciences clinical medicine hepatology
- medical and health sciences basic medicine physiology homeostasis
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Programme(s)
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Multi-annual funding programmes that define the EU’s priorities for research and innovation.
Topic(s)
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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
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Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
FP7-PEOPLE-2010-RG
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Funding Scheme
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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
75654 Paris
France
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.