Abstract: Thalassemia is a highly prevalent monogenic inherited hemolytic disease in southern China, typically caused by defects in the β-globin gene or α-globin gene that disrupt hemoglobin synthesis. China has over 30 million carriers of thalassemia genes, among whom about 300, 000 are patients with severe or intermediate forms. These patients rely on long-term blood transfusion to sustain their life, but iron overload can lead to multi-organ damage and premature death. Globally, approximately 400,000 newborns are diagnosed with hemoglobinopathies each year. At present, the only approved curative method in China is allogeneic hematopoietic stem cell transplantation, which faces limitations such as difficulties in matching donors, immune rejection, and high costs. In recent years, gene editing technology has brought breakthroughs for curing thalassemia. Several clinical trials of gene therapies based on autologous hematopoietic stem cells have demonstrated significant efficacy, enabling patients to become free from transfusion dependence. Current challenges mainly focus on long-term verification of technical safety, reducing production complexity, and cutting costs. With the future development of more precise gene editing technologies and the acceleration of clinical translation, gene therapy is expected to become a core curative solution for the one-time cure of thalassemia.