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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.

Call for proposal

Data not available
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Coordinator

ERASMUS UNIVERSITEIT ROTTERDAM
Address
Dr molewaterplein 50
3000 DR Rotterdam
Netherlands

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EU contribution
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Participants (6)