Objective The long term correction of sickle cell anaemia and thalassaemia by gene therapy requires the stable integration of a fully functional a- or beta-globin gene into the genome of a sufficient number of bone marrow (stem) cells of the patient. To achieve this two major problems will have to be addressed, namely stable delivery and expression. At present the efficient introduction and stable integration of genes into a host cell genome can be achieved with known retroviral and adeno associated viral (AAV) vectors. The most efficient elements to express a gene in a stable manner are LCR's. The objective of this proposal is to construct a LCR containing beta-globin transcription unit and viral vector technology that could lead to gene therapy clinical trials. As an attractive alternative we will also start the development of a non viral gene transfer system based on ligand mediated gene transfer. This system has the potential advantage that it has less size constraints and that it can be produced on large scale. Fields of science medical and health sciencesmedical biotechnologygenetic engineeringgene therapynatural sciencesbiological sciencesgeneticsgenomes Programme(s) FP4-BIOMED 2 - Specific research, technological development and demonstration programme in the field of biomedicine and health, 1994-1998 Topic(s) 5.4 - Somatic gene therapy Call for proposal Data not available Funding Scheme CSC - Cost-sharing contracts Coordinator Erasmus Universiteit Rotterdam EU contribution No data Address 50,Dr. Molewaterplein 3000 DR Rotterdam Netherlands See on map Total cost No data Participants (4) Sort alphabetically Sort by EU Contribution Expand all Collapse all National and Kapodestrian University of Athens Greece EU contribution No data Address 17,Aghiou Thoma Street 11527 Athens See on map Total cost No data Therexsys Ltd United Kingdom EU contribution No data Address ST5 5SP Keele See on map Total cost No data United Medical and Dental Schools of Guy's and St Thomas's Hospitals United Kingdom EU contribution No data Address London Bridge SE1 9RT London See on map Total cost No data Universita degli Studi di Cagliari Italy EU contribution No data Address Via Jenner s/n 09100 Cagliari See on map Total cost No data
