Abstract
Gene therapy can be used to restore cell function in monogenic disorders or to endow cells with new capabilities, such as improved killing of cancer cells, expression of suicide genes for controlled elimination of cell populations, or protection against chemotherapy or viral infection. While gene therapies were originally most often used to treat monogenic diseases and to improve hematopoietic stem cell transplantation outcome, the advent of genetically modified immune cell therapies, such as chimeric antigen receptor modified T cells, has contributed to the increased numbers of patients treated with gene and cell therapies. The advancement of gene therapy with integrating retroviral vectors continues to depend upon world-wide efforts. As the topic of this special issue is “Spotlight on Germany,” the goal of this review is to provide an overview of contributions to this field made by German clinical and research institutions. Research groups in Germany made, and continue to make, important contributions to the development of gene therapy, including design of vectors and transduction protocols for improved cell modification, methods to assess gene therapy vector efficacy and safety (e.g., clonal imbalance, insertion sites), as well as in the design and conduction of clinical gene therapy trials.
Highlights
Gene therapy is a molecular medicine approach that can be used to treat patients with inherited diseases, such as those caused by gene defects in monogenic diseases, as well as to treat acquired diseases, such as cancer and severe infections
A subsequent multinational study showed the efficacy and safety of a SIN gammaretroviral vector co-developed at Hannover Medical School to deliver the IL2RG gene to autologous bone marrow-derived CD34+ cells in nine boys with severe combined immunodeficiencies (SCIDs)-X1 (NCT01410019, NCT01175239, NCT01129544) [33]
In the first stem-cell gene therapy for Wiskott–Aldrich syndrome (WAS) patients conducted in Germany (German Clinical Trials Register number, DRKS00000330) [97, 98], ten patients were treated with autologous CD34+ hematopoietic stem and progenitor cells (HSPC) modified with an MLVderived long terminal repeats (LTR)-driven gammaretroviral vector (CMMP backbone—a derivative of MFG pseudotyped with gibbon ape leukemia virus (GALV)) engineered to express WASP
Summary
Gene therapy is a molecular medicine approach that can be used to treat patients with inherited diseases, such as those caused by gene defects in monogenic diseases, as well as to treat acquired diseases, such as cancer and severe infections. A subsequent multinational study showed the efficacy and safety of a SIN gammaretroviral vector co-developed at Hannover Medical School to deliver the IL2RG gene to autologous bone marrow-derived CD34+ cells in nine boys with SCID-X1 (NCT01410019, NCT01175239, NCT01129544) [33].
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