广州医药2024，Vol.55Issue(4)：331-341,359,12.DOI:10.3969/j.issn.1000-8535.2024.04.001
血友病基因治疗国内外研究新进展
Recent advancements in gene therapy for hemophilia
摘要
Abstract
Hemophilia is a genetic bleeding disorder resulting from mutations in coagulation factor genes on the X chromosome.The mainstay of current treatment is coagulation factor replacement therapy.However,frequent and long-term injections often lead to poor patient compliance,easy inhibitor development,and compromised therapeutic efficacy.Despite advancements in delivery methods and prolonged action of novel agents such as extended half-life coagulation factor concentrates,humanized bispecific antibodies,and anti-tissue factor pathway inhibitor monoclonal antibodies,these approaches still fall short of curing hemophilia.Consequently,gene therapy,aiming for disease eradication,has garnered significant attention in recent years.This review delves into the principles of gene therapy,the selection of gene therapy vectors,and gene therapy preconditioning regimens.It summarizes the safety and efficacy of gene therapy in current clinical applications and discusses challenges and future directions in this field.关键词
血友病/基因治疗/预处理方案/临床进展/存在问题Key words
hemophilia/gene therapy/conditioning/clinical advancements/challenges引用本文复制引用
李儒雅,梁亮,周睿卿,郝嘉懿,阎文锦,黄金棋,王顺清..血友病基因治疗国内外研究新进展[J].广州医药,2024,55(4):331-341,359,12.基金项目
国家自然科学基金(82270143) （82270143）
广东省基础与应用基础研究基金(2022A1515220122) （2022A1515220122）
湛江市科技计划项目(2022A01019) （2022A01019）
