The goal of this program project is to develop new and safe approaches for stem cell therapy in general and stem cell gene therapy for the beta-chain hemoglobinopathies, sickle cell anemia and homozygous beta-thalassemia, in particular. The program consists of six projects and four core units. The objective of Project by Stamatoyannopoulos is to develop new chromatin insulators for use in gene therapy vectors and investigate the functions of globin gene oncoretrovirus, foamy virus, lentivirus and Adeno AAV vectors in the primate transplantation model. Projects by Russell and Lieber focus on the development of new vector technologies for stem cell gene therapy; foamy viral vectors (Project by Russell); and deleted adeno AAV vectors containing the whole beta-globin gene locus control region (Project by Lieber). Success in the development of these new vectors will have a major impact on the field of stem cell gene therapy. Project by Blau uses a new technology, based on chemical inducers of dimerization, to achieve in vivo selection of genetically modified stem cells. Project by Kiem studies oncoretroviral and lentiviral vectors in the baboon model and attempts new approaches for improving stem cell engraftment. Project by Naldini focuses on the investigation of new approaches that will increase the efficiency and safety of lentiviral vectors. The NOD/SCID Mouse Core is a SCID/NOD mice unit and will assist investigators in the assessment of gene transfer into human hemopoietic stem cells. The Genomics Core is a genomic unit and will assist the projects with sequencing and functional analyses of viral vector investigation sites. The Large Animal Core will perform bone marrow transplantation in large animals (primates and dogs), to assess gene transfer into the hemopoietic stem cells. The Administration Core will provide administrative support.
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