Promising coagulation factor VIII bypassing strategies for patients with haemophilia A

被引：2
|
作者：
Duan, Xunwei [1 ,2 ]
Tang, Mingqing [1 ,2 ,3 ]
Zhang, Junping [1 ,2 ,3 ]
Yu, Hao [1 ,2 ]
Xu, Ruian [1 ,2 ,3 ]
机构：
[1] Huaqiao Univ, Sch Biomed Sci, Quanzhou, Fujian, Peoples R China
[2] Huaqiao Univ, Inst Mol Med, Quanzhou, Fujian, Peoples R China
[3] Minist Educ, Engn Res Ctr Mol Med, Xiamen 361021, Fujian, Peoples R China
来源：
BLOOD COAGULATION & FIBRINOLYSIS | 2014年 / 25卷 / 06期
基金：
国家高技术研究发展计划(863计划); 美国国家科学基金会;
关键词：
factor VIII; factor VIII bypassing; gene therapy; haemophilia A; neutralizing antibody; RECOMBINANT FACTOR VIIA; PROTHROMBIN COMPLEX CONCENTRATE; ACTIVATED FACTOR-VII; PROLONGED HALF-LIFE; FACTOR-X ACTIVATION; CELL-BASED MODEL; HUMAN-FACTOR-IX; BLOOD-COAGULATION; IN-VITRO; THROMBIN GENERATION;
D O I：
10.1097/MBC.0000000000000098
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
Haemophilia A is an X-linked recessive monogenic hereditary bleeding disorder caused by a deficiency or functional defect in coagulation factor VIII (FVIII). Typically, only 30% haemophilia A patients are treated with FVIII-specific products successfully. Therefore, other promising clotting factors and FVIII-bypassing factors exhibiting sufficient FVIII-independent activity, low immunogenicity and prolonged half-life are needed to conquer this malady. Here, we will systematically review the current status of the diverse FVIII-bypassing factors for the treatment of FVIII-insensitive haemophilia A patients. (C) 2014 Wolters Kluwer Health | Lippincott Williams & Wilkins.
引用
收藏
页码：539 / 552
页数：14
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