Liang Hao, Wang Yun-Xia, Li Xu-Yan, Wang Ya-Qi, Su Yan
Department of Biochemistry and Molecular Biology, Baotou Medical College, Baotou 014010, Inner Mongolia Autonomous Region, China.
Department of Biochemistry and Molecular Biology, Baotou Medical College, Baotou 014010, Inner Mongolia Autonomous Region, China,E-mail:
Zhongguo Shi Yan Xue Ye Xue Za Zhi. 2021 Apr;29(2):643-647. doi: 10.19746/j.cnki.issn.1009-2137.2021.02.054.
Sickle cell disease (SCD) is a single gene genetic disease, which seriously threatens the life span and quality of patients. On the basis of the pathogenesis of SCD and the alternative therapy based on fetal hemoglobin F (HbF), the research progress of transcription factors involved in the regulation of HbF gene expression, such as BCL11A, ZBTB7A, KLF-1, c-MYB and SOX6, as well as the application of CRISPR / Cas9, TALEN, zinc finger nuclease and other gene editing technologies in this field has been made, providing a solid theoretical basis for the exploration of new treatment schemes for β- like hemoglobin diseases, such as sickle cell disease and β- thalassemia.
镰状细胞病（SCD）是一种单基因遗传病，严重威胁患者的寿命和生活质量。基于SCD的发病机制以及基于胎儿血红蛋白F（HbF）的替代疗法，参与调控HbF基因表达的转录因子如BCL11A、ZBTB7A、KLF-1、c-MYB和SOX6的研究进展，以及CRISPR/Cas9、TALEN、锌指核酸酶等基因编辑技术在该领域的应用，为探索镰状细胞病和β地中海贫血等β类血红蛋白疾病的新治疗方案提供了坚实的理论基础。
