The Journal of Practical Medicine ›› 2021, Vol. 37 ›› Issue (6): 816-820.doi: 10.3969/j.issn.1006⁃5725.2021.06.026
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YANG Huamei*,SUN Fei,MA Yanlin,LI Qi.
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Abstract:
β⁃thalassemia,the most common form of thalassemia,is a monogenic inherited disease caused by the abnormality of β⁃globin gene. Although there are currently many treatments for thalassemia,such as blood transfusion,iron chelation,splenectomy,and allogeneic hematopoietic stem cell transplantation(HSCT),the pathway to cure thalassemia still faces great challenges. In this paper,the current clinical treatment strategies of β⁃thalassemia were systematically described based on the pathogenesis of the disease,and the research progress of how to cure β⁃thalassemia by gene therapy was emphatically described.
Key words: β?thalassemia, β?globin, γ?globin, lentiviral vector, gene therapy
YANG Huamei, SUN Fei, MA Yanlin, LI Qi..
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URL: https://www.syyxzz.com/EN/10.3969/j.issn.1006⁃5725.2021.06.026
https://www.syyxzz.com/EN/Y2021/V37/I6/816