Big stride in gene therapy for hemophilia B in China  

作　　者：Jianmin Wang Dan Yang 

机构地区：[1]Department of Hematology,Shanghai Changhai Hospital,Shanghai 200433,China [2]Shanghai Daopei Hospital for Hematological Disorders,LuDaoPei Medical Group,Shanghai 201112,China

出　　处：《Blood Science》2023年第2期138-139,共2页血液科学（英文）

摘　　要：In a recent issue of Lancet Haematology,Xue et al1 reported an adeno-associated virus(AAV)-based gene therapy in 10 patients with hemophilia B(HB)from China.BBM-H901,a novel vector comprised of an engineered liver-tropic AAV capsid(AAV843),synthesized liver-specific promoter and CpG reduced factor IX(FIX)Padua coding sequence,was infused in 10 patients(baseline FIX coagulation activity[FIX:C]were less than 2 IU/dL)after 1 week of prophylactic prednisone pretreatment(1 mg/kg per day).After a median follow-up of 58 weeks,mean FIX:C reached 36.9±20·5 IU/dL.No FIX inhibitors or serious adverse events were observed.All patients developed high titer neutralizing antibodies against vector capsid.The concentrations of alanine aminotransferase and aspartate aminotransferase in plasma were below the upper limit of normal range in 8 patients.No FIX concentrate infusion was needed after gene therapy for these patients.This is a huge step forward in the treatment of HB in China.

关 键 词：PATIENTS INFUSION TITER 

分 类 号：R554.1[医药卫生—血液循环系统疾病]