Progress in Gene Therapy of Sickle Cell Disease Based on Hemoglobin F--Review.
10.19746/j.cnki.issn.1009-2137.2021.02.054
- Author:
Hao LIANG
1
;
Yun-Xia WANG
1
;
Xu-Yan LI
1
;
Ya-Qi WANG
1
;
Yan SU
2
Author Information
1. Department of Biochemistry and Molecular Biology, Baotou Medical College, Baotou 014010, Inner Mongolia Autonomous Region, China.
2. Department of Biochemistry and Molecular Biology, Baotou Medical College, Baotou 014010, Inner Mongolia Autonomous Region, China,E-mail: synmg@126.com.
- Publication Type:Journal Article
- MeSH:
Anemia, Sickle Cell/therapy*;
Cell Line, Tumor;
DNA-Binding Proteins;
Fetal Hemoglobin/genetics*;
Genetic Therapy;
Humans;
Repressor Proteins/genetics*;
Transcription Factors
- From:
Journal of Experimental Hematology
2021;29(2):643-647
- CountryChina
- Language:Chinese
-
Abstract:
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.
