摘要
β地中海贫血(β地贫)是一种由β珠蛋白基因突变导致β珠蛋白合成障碍或结构改变而引起的常见遗传性血液病。当前针对β地贫的慢病毒载体(LVV)介导的基因治疗,主要分为利用LVV将具有完整功能的β-血红蛋白基因添加在染色体上的基因整合策略,以及利用LVV将特异性核酸酶递送到造血干细胞中,对β-珠蛋白(HBB)基因进行原位修复的基因组编辑策略。提高病毒滴度与转导效率、降低脱靶率及推进临床试验是这两种策略的主要研究方向。
β-thalassemia is a common monogenic inherited blood disorder caused by mutations ofβ-globin gene which results to synthesis obstacles or abnormal structure ofβ-globin.Gene therapy mediated by lentiviral vector(LVV)is divided into gene-integration strategies using LVV to add fully functionalβ-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.
作者
刘译泽
镡颖
朱宝生
LIU Yize;CHAN Ying;ZHU Baosheng(School of Medicine,Kunming University of Science and Technology,Kunming 650500;Department of Medical Genetics,the First People′s Hospital of Yunnan Province,the Affiliated Hospital of Kunming University of Science and Technology,Kunming 650032,China)
出处
《基础医学与临床》
2023年第12期1876-1880,共5页
Basic and Clinical Medicine
基金
国家自然科学基金地区基金(82060039)
云南省科技计划(202101AS070005)。
