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Results of a clinical study conducted by Fu Bin, deputy chief physician of the Department of Hematology, Xiangya Hospital, Central South University, in collaboration with the team of Liu Mingyao and Wu Yuxuan from a company in Shanghai, "CRISPR/Cas9-mediated BCL11A enhancer gene editing for the treatment of β0/β0 infusion-dependent β- Thalassemia", published as a monograph online in Nature Medicine (Q1, IF 8241) entitled "CRISPR/Cas9-mediated Gene Editing of the BCL11A Enhancer for Pediatric β0/β0 Transfusion-Dependent β-Thalassemia.
Thalassemia is a single-gene hereditary disease with the widest distribution and the largest number of people in the worl.
The conventional treatment methods for thalassemia major are standardized lifelong blood transfusion and iron removal therapy, which not only have limited efficacy and often lead to iron overload, but also have serious barriers to work, life and study, as well as difficulties in blood transfusion, serious consumption of blood resources, and social medical cost.
The study by Fu Bin and others from Xiangya Hospital of Central South University is the first successful implementation of thalassemia gene therapy in Asia, and the world's first successful study to reactivate gamma globin to treat β0/β0 thalassemia major children through CRISPR gene editing technolog.
After more than two years of preparatory work such as basic research and animal experiments, the research team launched this clinical study using gene editing technology to treat patients with β-thalassemia major in December 2019. After obtaining ethical approval, in May 2020, two children with severe β-thalassaemia were infused with autologous hematopoietic stem cells that had been gene-edited to reactivate γ-globin expressio.
Compared with β0/β+ type gene therapy, β0/β0 type patients have no expression of the two β genes at al.
It is reported that, as a clinical team focusing on the diagnosis and treatment of anemia diseases and rare diseases, since 2017, Fu Bin led the group to take the lead in carrying out hematopoietic stem cell transplantation for thalassemia major in Hunan Provinc.
