血友病基因治疗国内外研究新进展OA

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.