CONSERT is a consortium of European research and clinical institutions cooperating to develop gene therapy for inherited diseases and supported by the research programs of the Commission of the European Communities. CONSERT stands for “Concerted Safety and Efficiency Evaluation of Retroviral Transgenesis for Gene Therapy of Inherited Diseases”.
CONSERT accumulates the expertise of biomedical scientists who have a strong background in the development of all platform technologies required for successful gene therapy:
1. State-of-the-art design and production of retroviral and lentiviral gene transfer vectors
2. Basic and applied biology of hematopoiesis and cell transplantation
3. Relevant experimental models and clinical expertise for the treatment of inherited hematopoietic disorders
4. Diagnostic tools to evaluate the safety and efficiency of gene therapeutic interventions in preclinical models and clinical applications
CONSERT focuses on the design and preparation for clinical trial through research and preclinical evaluation of gene therapy for:
1. Severe combined immune deficiencies (SCID-X1, adenosine (ADA)-deficient SCID, recombination activating genes RAG1/RAG2 deficiencies)
2. Hemoglobinopathies and inherited anemias (Diamond-Blackfan anemia, Fanconi anemia, ß-thalassemia)
3. Wiskott-Aldrich Syndrome
4. Storage disorders (Metachromatic Leukodystrophy (MLD), Globoid Leukodystrophy (GLD), Gaucher disease)
5. Chronic Granulomatous Disease