Coagulation Factor IX for Hemophilia B Therapy

被引:27
作者
Orlova, N. A. [1 ,2 ]
Kovnir, S. V. [1 ,2 ]
Vorobiev, I. I. [1 ,2 ]
Gabibov, A. G. [1 ]
机构
[1] Russian Acad Sci, Shemyakin & Ovchinnikov Inst Bioorgan Chem, Moscow 117997, Russia
[2] Minist Healthcare & Social Dev Russian Federat, Hematol Res Ctr, Moscow 125167, Russia
关键词
coagulation factor IX; hemophilia B; heterologous protein expression systems; CLOTTING FACTOR-IX; RECOMBINANT FACTOR-IX; SUSTAINED PHENOTYPIC CORRECTION; PREVIOUSLY TREATED PATIENTS; MEDIATED GENE-TRANSFER; LONG-TERM CORRECTION; HUMAN PROTEIN-C; FACTOR-X; PHARMACOKINETIC PROPERTIES; STRUCTURAL-ANALYSIS;
D O I
10.32607/20758251-2012-4-2-62-73
中图分类号
Q5 [生物化学]; Q7 [分子生物学];
学科分类号
071010 ; 081704 ;
摘要
Factor IX is a zymogen enzyme of the blood coagulation cascade. Inherited absence or deficit of the IX functional factor causes bleeding disorder hemophilia B, which requires constant protein replacement therapy. Reviewed herein are the current state in the manufacturing of FIX, improved variants of the recombinant protein for therapy, transgenic organisms for obtaining FIX, and the advances in the gene therapy of hemophilia B.
引用
收藏
页码:62 / 73
页数:12
相关论文
共 50 条
[31]   Optimized human factor IX expression cassettes for hepatic-directed gene therapy of hemophilia B [J].
Zhang, Ru ;
Wang, Qiang ;
Zhang, Lin ;
Chen, Saijuan .
FRONTIERS OF MEDICINE, 2015, 9 (01) :90-99
[32]   Etranacogene dezaparvovec-drlb gene therapy for patients with hemophilia B (congenital factor IX deficiency) [J].
Sekayan, Tro ;
Simmons, Dana H. ;
von Drygalski, Annette .
EXPERT OPINION ON BIOLOGICAL THERAPY, 2023, 23 (12) :1173-1184
[33]   Membranous glomerulonephritis and nephrosis post factor IX infusions in hemophilia B [J].
Dharnidharka, VR ;
Takemoto, C ;
Ewenstein, BM ;
Rosen, S ;
Harris, HW .
PEDIATRIC NEPHROLOGY, 1998, 12 (08) :654-657
[34]   The novel mutation p.Asp315Tyr causes severe hemophilia B by impairing coagulation factor IX expression [J].
Lu, Zhengjing ;
Zhang, Huayang ;
Chen, Changming ;
Wu, Wenman ;
Wei, Hongying .
THROMBOSIS RESEARCH, 2021, 198 :23-25
[35]   The Function of extravascular coagulation factor IX in haemostasis [J].
Mann, David M. ;
Stafford, Katherine A. ;
Poon, Man-Chiu ;
Matino, Davide ;
Stafford, Darrel W. .
HAEMOPHILIA, 2021, 27 (03) :332-339
[36]   Emerging drugs for hemophilia B [J].
Mannucci, Pier Mannuccio ;
Franchini, Massimo .
EXPERT OPINION ON EMERGING DRUGS, 2014, 19 (03) :407-414
[37]   Safety of AAV Factor IX Peripheral Transvenular Gene Delivery to Muscle in Hemophilia B Dogs [J].
Haurigot, Virginia ;
Mingozzi, Federico ;
Buchlis, George ;
Hui, Daniel J. ;
Chen, Yifeng ;
Basner-Tschakarjan, Etiena ;
Arruda, Valder R. ;
Radu, Antoneta ;
Franck, Helen G. ;
Wright, J. Fraser ;
Zhou, Shangzhen ;
Stedman, Hansell H. ;
Bellinger, Dwight A. ;
Nichols, Timothy C. ;
High, Katherine A. .
MOLECULAR THERAPY, 2010, 18 (07) :1318-1329
[38]   Coronary artery bypass grafting in a patient with hemophilia B: continuous recombinant factor IX infusion as per the Japanese guidelines for replacement therapy [J].
Suzuki, Tomoyuki ;
Kawamoto, Shunsuke ;
Kumagai, Kiichiro ;
Adachi, Osamu ;
Kanda, Keisuke ;
Ishikawa, Masaaki ;
Okitsu, Yoko ;
Harigae, Hideo ;
Kurosawa, Shin ;
Saiki, Yoshikatsu .
GENERAL THORACIC AND CARDIOVASCULAR SURGERY, 2016, 64 (08) :481-483
[39]   Coronary artery bypass grafting in a patient with hemophilia B: continuous recombinant factor IX infusion as per the Japanese guidelines for replacement therapy [J].
Tomoyuki Suzuki ;
Shunsuke Kawamoto ;
Kiichiro Kumagai ;
Osamu Adachi ;
Keisuke Kanda ;
Masaaki Ishikawa ;
Yoko Okitsu ;
Hideo Harigae ;
Shin Kurosawa ;
Yoshikatsu Saiki .
General Thoracic and Cardiovascular Surgery, 2016, 64 :481-483
[40]   Effect of prepropeptide replacement on γ-carboxylation and activity of recombinant coagulation factor IX [J].
Vatandoost, Jafar ;
Bos, Mettine H. A. .
BIOTECHNOLOGY LETTERS, 2022, 44 (08) :975-984