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Content archived on 2024-05-21

Gene therapy of hematopoietic stem cells for inherited diseases

Objective

INHERINET aims to develop novel, safe and curative treatments based on therapeutic gene transfer into antilogous (i.e. patient-derived) haematopoietic stem cells for selected example hereditary diseases, which include immunodeficiency's, mucopolysaccharidosis VI, Diamond-Blackman anaemia and Gautier's disease. It entails:
1. State-of-the-art vector development and production.
2. Polyclinic engraftment of gene-modified cells in preclinical animal models and tolerance for transgenic products.
3. Selection of gene-modified cells.
4. Disease specific animal model s to evaluate safety and efficacy. The project is intended to conclude with the preparation of clinical protocols to file for clinical trials based on the achievements, enabling discussion with expert panels on social and ethical aspects of the proposed therapeutic intervention.

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.

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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.

Data not available

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.

CSC - Cost-sharing contracts

Coordinator

ERASMUS UNIVERSITEIT ROTTERDAM
EU contribution
No data
Address
Dr Molewaterplein 50
3000 DR ROTTERDAM
Netherlands

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Total cost

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.

No data

Participants (6)

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