Science: crispr-cas9 editor car-t cell therapy can be used to treat refractory cancer

February 8, 2020 / Biovalley BIOON / -- according to the latest data released by researchers from the Abramson cancer center at the University of Pennsylvania, car-t cells edited by crispr-cas9 gene can last for several months in cancer patients, and can stably proliferate and function The study is the first clinical trial to test human gene editing methods in the United States In a recent article published in science, the researchers introduced the results of phase I clinical trial of gene editing car-t treatment for three patients "The data we got from three patients demonstrates two important things First of all, we can edit many times accurately and successfully in the manufacturing process; second, up to now, these cells have shown the ability to continuously attack and kill tumors, "said Professor Carl June, senior author of the article The discovery is another achievement of the University of Pennsylvania as a pioneer in cell and gene therapy, which has previously developed the first FDA approved car-t cell therapy kymriah for children and adults with blood cancer (photo source: Joseph fraietta, PhD, University of Pennsylvania) the methods in this study are closely related to car T-cell therapy In traditional car T-cell therapy, patients' immune cells are genetically modified to fight cancer But the method of this experiment is different Like car T, the researchers in this study first collected T cells from patients' blood However, the researchers did not transfer any car receptor molecules (such as CD19) targeting cancer cell antigens to these T cells, but first removed three key genes using CRISPR / cas9 editing Two of these genes express the natural receptors of T cells, namely TCRA and TCRB genes, while the third gene expresses PD-1, which is considered to be the key "checkpoint" molecule regulating T cell activity "This new analysis of three patients has confirmed the ability of CRISPR / cas9 to target multiple human genes at the same time by making cells that contain all three kinds of editing." Once these three genes are knocked out, the fourth gene modification can be completed by using lentivirus to insert cancer specific synthetic T cell receptor The modified T cell can target an antigen called NY-ESO-1 Compared with the original car-t cells in vivo, the new analysis shows that the edited cells can last for up to nine months A few months after reinfusion, the researchers took more blood and isolated CRISPR edited cells for research The results show that these cells still have the ability to kill tumors "Previous studies have shown that these cells lose their function within a few days, so the fact that CRISPR editing cells in this study can maintain their anti-tumor function for a longer time after a single infusion is encouraging," June said Sources of information: CRISPR edited immune cells can survive and deliver after information into cancer patients Original sources: E.A stadtmauer El Al., "CRISPR engineered T cells in patients with referral cancer," Science (2020) Science Sciencemag.org/lookup/ 1126/science.aba7365