Objective
We have developed the tools and experimental models and have provided the proof of principle that gene modified autologous haematopoietic stem cells can restore a complete immunological system in vivo in a particular form of SCID. We are pursuing these efforts applying this strategy to several other diseases : two lentivirus protocols for X-linked adrenoleukodystrophy, Thalassemia and sickle cell disease, are developed in our Clinical Investigation Centre. In 2010, we plan to initiate two other gene therapy trials for two primary immunodeficiencies (SCID-X1 and Wiskott-Aldrich syndrome). Our expertise in translational medicine has now been used to transfer into clinics a new gene therapy protocol for a devastating inherited skin disease : dystrophic epidermolysis bullosa. To complement these approaches, we optimize HLA-mismatched haematopoietic stem cell transplantation (HSCT). They are an important therapeutic option for children with primary immunodeficiencies but the delayed reconstitution of the T-cell compartment following T-cell depleted HSCT remains a major clinical concern thus drastically limiting its broader clinical application. In order to speed-up the immunological reconstitution in this setting, we try to pre-clinically develop a new approach able to generate a large quantity of T-cellular precursors from a fraction of the donor CD34+ stem cells shortly cultured on Notch delta 4 ligand. This strategy could ideally provide the recipient with a pool of diverse T-cells within the first month after transplantation conferring T-cellular immunity before de novo thymopoiesis takes place.
The results obtained with this procedure are so important that we think to extend its use to the transduction of HSC for the gene therapy trials.
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.
- medical and health sciences medical biotechnology genetic engineering gene therapy
- medical and health sciences medical biotechnology cells technologies stem cells
- medical and health sciences clinical medicine transplantation
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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-2010-AdG_20100317
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
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.