Lentivirus vector in gene therapy of β-thalassemia
10.16352/j.issn.1001-6325.2023.12.1876
- VernacularTitle:慢病毒载体在β地中海贫血基因治疗中的研究进展
- Author:
Yize LIU
1
;
Ying CHAN
;
Baosheng ZHU
Author Information
1. 昆明理工大学 医学院,云南 昆明 650500
- Keywords:
lentivirus vector;
gene therapy;
β-thalassemia
- From:
Basic & Clinical Medicine
2023;43(12):1876-1880
- CountryChina
- Language:Chinese
-
Abstract:
β-thalassemia is a common monogenic inherited blood disorder caused by mutations of β-globin gene which results to synthesis obstacles or abnormal structure of β-globin.Gene therapy mediated by lentiviral vector(LVV)is divided into gene-integration strategies using LVV to add fully functional β-hemoglobin(HBB)genes to chromosomes and gene-editing strategies using LVV to deliver specific ribozymes to hematopoietic stem cells for in situ repair of HBB genes.Improving viral titration and transduction efficiency,reducing target loss and advancing clinical trials are the main targets of these two strategies.
