Institute for Transfusion Medicine and Gene Therapy

Many congenital diseases of the blood system, such as severe combined immunodeficiency (SCID), are caused by well-characterized genetic defects. The standard therapy for most of these diseases is allogeneic stem cell transplantation (allo-SCT), however not for all of these patients an HLA-matched stem cell donor can be found. To help patients without a matching donor, we develop methods that allow us to genetically correct the patient's own blood stem cells, such that these cells can be used in an autologous transplantation setting. In order to increase the safety of gene therapy, we have established precise gene editing which allows us to correct the genome defect of human cells in a targeted fashion using highly specific molecular scissors, such as TALENs or CRISPR/Cas9-based nucleases.

Based on many years of experience with the transplantation of blood stem cells, the combination of stem cell therapy with gene therapy is a very promising approach for the treatment of both primary and acquired immunodeficiencies. To this end, the Institute for Cell and Gene Therapy cooperates with clinically oriented groups within the Center for Chronic Immunodeficiency (CCI) to successfully apply the resulting translational research findings to the clinical setting. Our research efforts focus on the production of gene therapeutics for the treatment of hemophagocytic lymphohistiocytosis (HLH), Hyper-IgE-Syndrome (HIES) and HIV infection.