Institute for Transfusion Medicine and Gene Therapy

The research activities at the Institute for Transfusion Medicine and Gene Therapy focus on the development and evaluation of safe approaches for the manufacturing of stem cell products and therapeutically active immune cells using modern genome editing platforms. Based on many years of clinical experience in transplanting hematopoietic cells, such as T cells and hematopoietic stem cells, the combination of cell and gene therapy is a particularly promising approach for restoring the function of a defective immune system in patients with acquired or inborn errors of immunity.

The three major goals of the R&D department are

• to further improve safety and efficacy genome editing tools (incl. TALENs, CRISPR/Cas9) for therapeutic applications in immune cells or hematopoietic stem cells
• to develop disease models and cell therapies based on multipotent und pluripotent stem cells, and
• to translate cell and gene therapy efforts into the clinic

To achieve these goals, the Institute for Transfusion Medicine and Gene Therapy has established numerous collaborations with clinically oriented research teams at the Center for Chronic Immunodeficiency (CCI) and the Comprehensive Cancer Center Freiburg (CCCF). In the mid-term, gene therapeutics are planned for the treatment of congenital defects of the immune system, for treatment of HIV infection and for combating prostate cancer.

Another research focus of the institute is the targeted genome modification in induced pluripotent stem cells (iPS cells) to establish cell-based disease models. Patient-derived iPS cells are increasingly important in regenerative medicine because iPS cells can – at least theoretically – be differentiated into all types of tissues after successful gene correction. An important development is the targeted differentiation of iPS cells into transplantable stem cells and effector cells. In collaboration with various research partners, we are establishing protocols to achieve targeted differentiation of these stem cells into functional B and T lymphocytes as well as monocytes and granulocytes.

An immunogenetic research focus is the characterization of Human Leukocyte Antigen (HLA)-mediated signaling pathways and the targeted modification of individual HLA genes. The latter will be the basis for the production of universally immunologically compatible cells for transplantation.