China News Service, Kunming, January 6th (Chen Jingshi, Shi Zhou Li Shiqi) The reporter learned from the 920 Hospital of the Joint Service Forces of the Chinese People's Liberation Army (hereinafter referred to as 920 Hospital) on the 6th that the first case of "HBB gene repaired thalassemia major in China" The clinical research of gene therapy has achieved initial success in the Department of Hematology of the hospital.
By repairing the HBB gene of patients with thalassemia major, the patient successfully restored normal hematopoietic function and got rid of blood transfusion. This is the first case in the country and it is expected to completely cure thalassemia major.
In recent years, the team of Wang Sanbin, director of the Department of Hematology of 920 Hospital, has been committed to exploring the radical cure of some complex blood diseases through cell therapy and gene therapy.
12-year-old Pingping (a pseudonym) was diagnosed with thalassemia major at 6 months of birth. In the absence of conditions for receiving hematopoietic stem cell transplantation, Pingping’s family had to choose a blood transfusion every 20 days to maintain life, and it lasted for ten. year.
In September 2021, after Pingping's family learned that 920 Hospital is expected to cure thalassemia major without allotransplantation, they rushed from Qianxinan Prefecture, Guizhou Province to Kunming City, Yunnan Province for treatment.
Under the leadership of Wang Sanbin, the Department of Hematology of the hospital implemented the country's first thalassemia major gene therapy with HBB gene repair for Pingping.
Through clinical observations nearly 4 months after treatment, it was found that the gene-repaired cells gradually recovered stable hematopoietic function, and Pingping's palms and soles gradually became bloody, her physical condition gradually improved, and she has initially got rid of blood transfusion.
"My child is getting better at last! I should be able to go home for the New Year in a while." Pingping's mother said excitedly.
Wang Sanbin introduced, “920 Hospital is currently cooperating with the team of Professor Cai Yujia of Shanghai Jiaotong University to carry out gene therapy for β-thalassemia major. Through years of research and the results of this treatment, the safety and effectiveness of the technology have been preliminarily confirmed. "Next, Wang Sanbin will lead the team to deepen research and continue to cooperate with scientific research institutions and charities to make this technology benefit more patients.
The initial success of this treatment announced a major breakthrough in the first domestic gene therapy of thalassemia major with HBB gene repair.
It is expected to achieve formal clinical application in the future, which will revolutionize the treatment of thalassemia major.
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