Research Progress on Gene Therapy for β-thalassemia---Review.
10.19746/j.cnki.issn.1009-2137.2021.05.050
- Author:
Wei-Cong HONG
1
;
Jian-Pei FANG
2
;
Lyu-Hong XU
1
Author Information
1. Department of Pediatrics, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou 510000, Guangdong Province, China.
2. Department of Pediatrics, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou 510000, Guangdong Province, China E-mail: jpfang2005@163.com.
- Publication Type:Review
- MeSH:
Gene Editing;
Genetic Therapy;
Genetic Vectors;
Humans;
beta-Globins/genetics*;
beta-Thalassemia/therapy*
- From:
Journal of Experimental Hematology
2021;29(5):1676-1679
- CountryChina
- Language:Chinese
-
Abstract:
β-thalassemia is a monogenetic inherited hemolytic anemia, which results in a series of pathophysiological changes due to partial or complete inhibition of the synthesis of β-globin chain. The curative therapy for this disease is to reconstitute hematopoiesis, and transplantation with genetically modified autologous hematopoietic stem cells can avoid the major difficulties of traditional allogeneic hematopoietic stem cell transplantation，such as HLA matching and immune rejection. β-thalassemia gene therapy strategies mainly include gene integration and genome editing. The former relies on the development of lentiviral vectors and adds a fully functional HBB gene to the chromosome; the latter rapidly develops with the research of specific nuclease which can repair the HBB gene in situ. In this review, the latest progress of the two strategies in gene therapy of β-thalassemia is summarized.
