A TEAM of medical researchers in Shanghai has demonstrated that gene-editing technology can effectively treat thalassemia, a hereditary blood disorder, potentially offering patients a one-time cure. According to media reports, the team led by Zhai Xiaowen, deputy director of the Children's Hospital of Fudan University, and Chen Jia from ShanghaiTech University, has successfully treated multiple children with thalassemia using gene therapy over the past two years. Unlike common anemia, which is often caused by malnutrition or blood loss, thalassemia results from a genetic defect that impairs hemoglobin synthesis, leading to chronic anemia. In China, there are over 30 million carriers of the thalassemia gene, primarily in Guangdong and Hainan provinces and the Guangxi Zhuang Autonomous Region, with tens of thousands of severe cases reported. Severe thalassemia poses a significant threat to life. Patients typically require lifelong regular blood transfusions. Delayed transfusions can lead to tissue hypoxia, heart failure, or multi-organ damage. Conventional treatment for severe thalassemia relies on allogeneic hematopoietic stem cell transplantation, which faces challenges such as donor matching difficulties, high surgical risks, and potential post-operative rejection. In contrast, gene therapy involves harvesting the patient’s own stem cells, editing them in the laboratory, and reinfusing them into the patient. According to Zhai, this approach significantly reduces surgical risks and eliminates the need for a donor. "After the procedure, patients require far fewer blood transfusions — some even stop needing them entirely," Zhai stated. "This fundamentally improves their quality of life and confirms the efficacy of gene-editing treatment." The research team will conduct a 15-year follow-up study to evaluate the long-term safety and effectiveness of the therapy. They also plan to recruit international patients to receive free treatment. Zhai noted that gene-editing technology is scalable and holds promise for treating other rare blood disorders beyond thalassemia. He added that future efforts will focus on leveraging artificial intelligence to predict treatment outcomes and mitigate risks for individual patients. (SD-Agencies) | 