期刊
GENE THERAPY
卷 28, 期 9, 页码 494-512出版社
SPRINGERNATURE
DOI: 10.1038/s41434-021-00237-x
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类别
资金
- Deutsche Forschungsgemeinschaft [SFB841, SFB738]
- REBIRTH Center for Translational Regenerative Medicine through the State of Lower Saxony [MWK: ZN3440]
- Deutsche Krebshilfe
- Comprehensive Cancer Center (CCC) Hannover
- European Union [755170, 666908]
- European Research Council (ERC) [819531]
- Deutsche Forschungsgemeinschaft (Cluster of Excellence REBIRTH (EXC 62/2))
- H2020 Societal Challenges Programme [755170] Funding Source: H2020 Societal Challenges Programme
- European Research Council (ERC) [819531] Funding Source: European Research Council (ERC)
Gene therapy can be used to treat monogenic disorders or enhance cells with new abilities. German research institutions have made significant contributions to the development of gene therapy, including designing vectors, conducting clinical trials, and assessing vector efficacy and safety.
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.
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