CONSERT will integrate leading European activities for a structured implementation of novel therapies using genetically enhanced postnatal stem cells, with focus on the treatment of monogenic immunodeficiencies, hemoglobinopathies, anemias and storage disorders. CONSERT will develop and evaluate methods for genetic stem cell modification with wide implications for many other disorders including viral infection and cancer. A central theme of the project is an unbiased safety and efficiency evaluation of the key technology used in the genetic modifications of replicating somatic cells: retroviral vector-mediated transgenesis. This is only possible through concerted multi-center studies. Lenti-, spuma- and gamma- retroviral vectors will be tailor-made for target disorders and tested for potency and safety in preclinical disease models. Designed with a translational aim, basic studies in stem cell biology and selectable marker technology will complement this research. A most important aspect of CONSERT is the molecular and clinical monitoring of currently active and success- ful clinical trials of genetic therapies. This will create a paradigmatic data-mining activity to obtain insights into crucial issues of clonal kinetics of gene-modified cells in vivo. Molecular studies in precise cell systems and animal models will provide the mechanistic understanding of transgene-host interactions. This project will generate the basis for technology development and promote patient safety. Translational dissemination of know-how from academia to industry will create a network of cell processing manufacturers with large economic potential, and prepare future clinical studies with improved predictability. Supervised by a proactive ethical project, CONSERT will stimulate a competitive and complementary evolution of biomedical academia, clinics, innovative health service providers, and associated training opportunities.
Keywords:
Inherited diseases, stem cell biology, gammaretrovirus, lentivirus, immunodeficiencies, red cell disorders, storage diseases, Wiskott-Aldrich syndrom, leukodystrophies, chronic granulomatour disease
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