Three young patients with relapsed T-cell leukemia have now been treated with base-edited T-cells, as part of a 'bench-to-bedside' collaboration between UCL and Great Ormond Street Hospital for Children (GOSH).
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Three young patients with relapsing T-cell leukemia have now been treated with CAR T cells. Data from the clinical trial show how the donor CAR T cells were engineered using cutting-edge gene-editing technology to modify single letters of their DNA code so that they could fight leukemia. To generate "universal" anti-T CAR T cell banks for the study, the researchers used T cells from healthy donors. They then made modifications to the cells using basic editing. The steps were: the deletion of existing receptors so that a donor's T cells could be banked and used without pairing, making them "universal", the deletion of a "flag" called CD7 so that T cells would not kill each other, the deletion of a second "flag" called CD52 to make the edited cells invisible to some of the potent drugs administered to the patient during the treatment process, and the addition of a chimeric antigen receptor that recognizes the CD7 T-cell receptor on leukemic T-cells to combat T-cell leukemia. The clinical trial for this treatment is still open and aims to recruit up to 10 patients. The teams hope that the treatment can be offered earlier in the treatment pathway. With additional funding, they also hope to make it available for adults in the future.Â