Objective
Liver diseases are becoming one of the most common causes of mortality in developing countries, and orthotopic liver transplantation is the only available treatment. However, this procedure carries implies indefinite immunosupression treatment associated with heavy side effects and eventual rejection. Furthermore, an increasing number of patients die while on the liver transplant waiting list due to the shortage of donor livers. Hepatocyte transplantation recently became an alternative to transplantation for the treatment of life-threatening Ineherithed Metabolic Diseases (IMDs) of the liver. However, this approach is also hampered by the lack of donors and by the difficulty in expending hepatocytes in vitro. Therefore, developing alternative source of hepatocytes represents a major challenge for the regenerative medicine field. Pluripotent stem cells generated from reprogrammed somatic cells (human Induced Pluripotent Stem Cells or hIPSCs) represent an advantageous solution since they can proliferate indefinitely in vitro while maintaining their capacity to differentiate into a broad number of cell types including hepatocytes. In addition, hIPSCs could enable the production of patient specific cells fully immuno-compatible with the original donor thereby avoiding the need for immune suppressive treatment. Here, we propose to systematically address the limitations preventing the use of hIPSCs for cells based therapy of IMDs. We will first develop novel methods to generate “better” hIPSCs fully compatible with clinical applications and to differentiate them into adult hepatocyes. In parallel, we will establish a novel approach for editing the mammalian genome and to correct the genetic defects associated with IMDs. Finally, we will validate the safety and the functionality of hIPSCs derived hepatocytes in vivo. Overall this comprehensive study will aim to provide the first proof of principle that hIPSCs could be useful in novel therapies against IMDs.
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.
- social sciences sociology demography mortality
- medical and health sciences medical biotechnology cells technologies stem cells
- medical and health sciences clinical medicine hepatology
- medical and health sciences clinical medicine transplantation
- natural sciences biological sciences genetics genomes
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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.
ERC-2011-StG_20101109
See other projects for this call
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.
Host institution
CB2 1TN Cambridge
United Kingdom
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.