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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.

Funding Scheme

CSC - Cost-sharing contracts

Coordinator

ERASMUS UNIVERSITEIT ROTTERDAM
Address
Dr Molewaterplein 50
3000 DR Rotterdam
Netherlands

Participants (6)

ALBERT-LUDWIGS-UNIVERSITAET FREIBURG
Germany
Address
Fahnenbergplatz
79085 Freiburg (In Breisgau)
GENETHON III
France
Address
Rue De L'internationale 1Bis
91002 Evry
HANNOVER MEDICAL SCHOOL
Germany
Address
Carl-neuberg-strasse 1
30625 Hannover
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
France
Address
Rue De Sèvres 149
75743 Paris
LUND UNIVERSITY
Sweden
Address
Paradisgatan 2
221 00 Lund
SERVEIS SANITARIS DE REFERENCIA - CENTRE DE TRANFUSIO I BANC DE TEIXITS
Spain
Address
Pg. Vall D'hebron 119-129
08035 Barcelona