According to Guangxi Medical University, the Mediterranean anemia prevention and treatment team of the First Affiliated Hospital of the university, together with Shanghai University of Science and Technology, Fudan University, and Zhengxu Biology, has cured 5 patients with β - thalassemia through base editing therapy. The relevant clinical application results have been published in the international journal Nature. Mediterranean anemia is a congenital hereditary blood disorder. Lai Yongrong, Deputy Director of the Mediterranean Anemia Prevention and Control Key Laboratory (Co built) of the National Health Commission and Deputy Director of the Mediterranean Anemia Prevention and Control Research Institute of Guangxi Medical University, introduced that currently, although allogeneic hematopoietic stem cell transplantation is recognized as a curative method, it has significant limitations in clinical application due to age restrictions and high risk of complications. The breakthrough of gene editing technology has opened a new door for the cure of thalassemia. In 2023, the First Affiliated Hospital of Guangxi Medical University launched this independently developed and world first clinical trial of base editing therapy in China, relying on CS-101 injection for a 3-year clinical study. We first extract the patient's own hematopoietic stem cells, perform precise single base editing on the target gene region in vitro, reactivate the target globin, and then transfuse the edited hematopoietic stem cells back into the patient's body to help them rebuild normal hematopoietic function, "explained Lai Yongrong. The experimental results showed that after receiving a single edited hematopoietic stem cell infusion, the hematopoietic function of 5 patients with β - thalassemia was rapidly rebuilt. The median time for neutrophil implantation was only 16 days, and the total hemoglobin level returned to normal. All patients successfully got rid of transfusion dependence, and no serious adverse reactions occurred during the median follow-up of 23 months. The reviewers of Nature evaluated the efficacy and safety of this therapy, setting a "new high" for in vitro edited hematopoietic stem cell therapy for β - thalassemia. This achievement has opened up a new path for the cure of hereditary blood diseases and provided important references for the prevention and treatment of major diseases such as tumors. ”Liu Rongrong, Deputy Director of the Mediterranean Anemia Prevention and Control Key Laboratory (Co built) of the National Health Commission and Deputy Director of the Hematology Department of the First Affiliated Hospital of Guangxi Medical University, said. (Looking into the New Era)