Single cell analysis of lentiviral integration to support ex-vivo gene modified cell therapy development

      Ex-vivo gene modified cell therapies are increasingly being developed for the treatment of monogenic diseases and various forms of cancer. In the last few years three gene modified cell therapy products have been licenced by the FDA or EMA and there are currently over 600 clinical trials being conducted using this type of cell therapy. Of particular interest are immunotherapies targeting haematological pathologies, due to the easy retrieval of peripheral blood mononuclear cells from patients and the range of viral vectors available for their ex vivo manipulation. However, the risks associated with therapies based on genetically modified cells are not fully documented and safety concerns could hamper their ongoing development.
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