The research team for the prevention and treatment of thalassemia at the First Affiliated Hospital of Guangxi Medical University, in collaboration with ShanghaiTech University, Fudan University, and Zhengxu Biotechnology, has successfully completed the world's first clinical application of base edited drugs for the treatment of transfusion dependent beta thalassemia. The relevant results were recently published in the journal Nature. The study was led by Professor Lai Yongrong and Professor Liu Rongrong from the Hematology Department of the First Affiliated Hospital of Guangxi Medical University. The related results have brought a new one-time cure plan for patients with severe β - thalassemia. β - thalassemia is a severe genetic blood disease that is prevalent in some southern regions of China. ”Lai Yongrong introduced, "For transfusion dependent patients, in the past, they could only rely on lifelong regular blood transfusions to maintain their lives. They not only faced problems such as blood supply shortage and organ damage caused by iron overload in the body, but also had to rely on allogeneic hematopoietic stem cell transplantation as the only cure. Due to the difficulty of matching and the risk of rejection reactions, most patients missed the opportunity for treatment." The breakthrough of this research team is the use of safer and more accurate base editing technology. By directly rewriting the wrong base on the gene, the pathogenic gene can be accurately repaired, activating the human body's normal working fetal hemoglobin and compensating for defective hemoglobin. Safety and accuracy have been greatly improved. In the clinical trial, 5 patients with transfusion dependent beta thalassemia received relevant treatment. Clinical data shows that all patients have rapid recovery of hematopoietic function after treatment, achieving hematopoietic cell implantation as early as 14 days. All patients successfully get rid of lifelong blood transfusion within one month, and their hemoglobin levels steadily rise to the normal range and remain stable for a long time. As of now, the first patient has been separated from blood transfusion for more than 28 months, and there have been no serious treatment-related adverse reactions during the follow-up period. Precise testing has also not found any off target gene editing phenomenon. (New Society)