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

• medical and health sciences › medical biotechnology › genetic engineering › gene therapy
• medical and health sciences › medical biotechnology › cells technologies › stem cells

Programme(s)

• FP5-LIFE QUALITY - Specific Programme for research, technological development and demonstration on "Quality of life and management of living resources", 1998-2002

Topic(s)

• 1.1.1.-3. - Key action The "Cell factory"

Call for proposal

Data not available

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