Objective
Research objectives and content
Somatic gene therapy constitute an alternative strategy to Bone Marrow Transplantation in order to cure primary immunodeficiencies, such as Chronic Granulomatous Disease. The optimal target cell population for such a strategy is the pluripotent hematopoietic stem cell, which is defined by the capacity for extensive self-renewal, retention of multilineage differentiation potential and long term reconstitution of entire haematopoietic system. Nevertheless successes in mouse model, retroviral-mediated gene transfer into a significant proportion of human hematopoietic repopulating cells using vectors based on the Moloney murine leukemia remains inefficient.
To overcome some of these problems, different pseudotyped retroviral vectors, such as the feline endogenous virus | or the gibbon ape leukemia virus, will be tested to transduce human stem cell from cord blood and bone marrow | cells. The ability to efficiently transduce these cells will be tested using an in vivo functional assay for primitive . human hematopoietic cells based on their ability to repopulate bone marrow of immunodeficient nonobese . diabetic/severe combined immunodeficient mice..
Because defect genes, such as P47phox, implicated in Chronic Granulomatous Disease are expressed in myeloid . cells, the second objective is to achieve optimal expression of the transgene in the mature myeloid cells. We will test optimised retroviral regulatory sequences to achieve this goal. Training content (objective, benefit and expected impact)
.
The participation of the proposed project would allow me to expand my knowledge on gene therapy, haematology I and in vivo mouse model. Because a number of gene therapy programmeare under-development in Europe, this ( research experience at the Institute of Child Health which presents biology and medical interface, will constitute a significant advantage for the future.
Links with industry / industrial relevance (22)
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 hematology
- medical and health sciences clinical medicine transplantation
- medical and health sciences clinical medicine oncology leukemia
You need to log in or register to use this function
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.
Data not available
Procedure for inviting applicants to submit project proposals, with the aim of receiving EU funding.
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.
Coordinator
WC1N 1EH London
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.