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
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