Presented in collaboration with Be The Match BioTherapies®
Induced pluripotent stem cells (iPSCs), derived from adult cells, have the potential to form any cell type in the body. iPSCs are promising research tools for studying human development, modeling disease and potentially treating patients. Hemoglobinopathies such as sickle cell disease (SCD) and beta thalassemia (BT) are treated with a limited number of pharmacologic options and blood transfusions. The only curative treatment modality is allogeneic transplantation and autologous transplantation with hematopoietic progenitor cell (HPC)-based gene therapy but these approaches have limitations and the latter is still in clinical trials. HPC-based approaches require obtaining sufficient numbers of HPCs via a bone marrow harvest or peripheral blood stem cell collection and maintaining the HPCs ex vivo for more than a short period of time for ex vivo manipulation without a loss of engraftment ability which are challenging to do. iPSCs may offer potential benefits over HPC-based approaches. This session will provide information on the current state of iPSCs for the treatment of hemoglobinopathies.
Discuss the role of iPSCs for drug screening and disease modeling
Discuss erythroid cell generation from iPSCs obtained from individuals without hemoglobinopathies and their potential clinical applications
Discuss cell and gene therapy for beta hemoglobinopathies with iPSCs