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

来源：

ACTA NATURAE | 2012年 / 4卷 / 02期

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

页码：62 / 73

页数：12

共 50 条

[21] Structural and functional exploration of three newly identified coagulation factor IX mutations in Chinese hemophilia B patients [J].

Wang, Rui ;

Jiang, Shifeng ;

Wang, Xuefeng ;

Wei, Dongqing ;

Xu, Qin ;

Wu, Wenman .

INTERNATIONAL JOURNAL OF HEMATOLOGY, 2023, 118 (02) :201-209

[22] Population pharmacokinetics of a new long-acting recombinant coagulation factor IX albumin fusion protein for patients with severe hemophilia B [J].

Zhang, Y. ;

Roberts, J. ;

Bensen-Kennedy, D. ;

Jacobs, I. ;

Santagostino, E. ;

Voigt, C. ;

Feussner, A. ;

Morfini, M. ;

Sidhu, J. .

JOURNAL OF THROMBOSIS AND HAEMOSTASIS, 2016, 14 (11) :2132-2140

[23] Transforming the treatment for hemophilia B patients: update on the clinical development of recombinant fusion protein linking recombinant coagulation factor IX with recombinant albumin (rIX-FP) [J].

Santagostino, Elena .

THROMBOSIS RESEARCH, 2016, 141 :5-8

[24] Factor VIII and Factor IX Activity Measurements for Hemophilia Diagnosis and Related Treatments [J].

Bowyer, Annette E. ;

Gosselin, Robert C. .

SEMINARS IN THROMBOSIS AND HEMOSTASIS, 2023, 49 (06) :609-620

[25] Biodistribution of recombinant factor IX, extended half-life recombinant factor IX Fc fusion protein, and glycoPEGylated recombinant factor IX in hemophilia B mice [J].

van der Flier, Arjan ;

Hong, Vu ;

Liu, Zhan ;

Piepenhagen, Peter ;

Ulinski, Gregory ;

Dumont, Jennifer A. ;

Orcutt, Kelly D. ;

Goel, Apollina ;

Peters, Robert ;

Salas, Joe .

BLOOD COAGULATION & FIBRINOLYSIS, 2023, 34 (06) :353-363

[26] Persistent expression of canine factor IX in hemophilia B canines [J].

H Chao ;

R J Samulski ;

D A Bellinger ;

P E Monahan ;

T C Nichols ;

C E Walsh .

Gene Therapy, 1999, 6 :1695-1704

[27] Persistent expression of canine factor IX in hemophilia B canines [J].

Chao, H ;

Samulski, RJ ;

Bellinger, DA ;

Monahan, PE ;

Nichols, TC ;

Walsh, CE .

GENE THERAPY, 1999, 6 (10) :1695-1704

[28] Complete deletion of factor IX gene and inhibition of factor IX activity in a Labrador Retriever with hemophilia B [J].

Brooks, MB ;

Gu, WK ;

Ray, K .

JOURNAL OF THE AMERICAN VETERINARY MEDICAL ASSOCIATION, 1997, 211 (11) :1418-&

[29] Phase 3 Study of Recombinant Factor IX Fc Fusion Protein in Hemophilia B [J].

Powell, Jerry S. ;

Pasi, K. John ;

Ragni, Margaret V. ;

Ozelo, Margareth C. ;

Valentino, Leonard A. ;

Mahlangu, Johnny N. ;

Josephson, Neil C. ;

Perry, David ;

Manco-Johnson, Marilyn J. ;

Apte, Shashikant ;

Baker, Ross I. ;

Chan, Godfrey C. ;

Novitzky, Nicolas ;

Wong, Raymond S. ;

Krassova, Snejana ;

Allen, Geoffrey ;

Jiang, Haiyan ;

Innes, Alison ;

Li, Shuanglian ;

Cristiano, Lynda M. ;

Goyal, Jaya ;

Sommer, Jurg M. ;

Dumont, Jennifer A. ;

Nugent, Karen ;

Vigliani, Gloria ;

Brennan, Aoife ;

Luk, Alvin ;

Pierce, Glenn F. .

NEW ENGLAND JOURNAL OF MEDICINE, 2013, 369 (24) :2313-2323

[30] Optimized human factor IX expression cassettes for hepatic-directed gene therapy of hemophilia B [J].

Ru Zhang ;

Qiang Wang ;

Lin Zhang ;

Saijuan Chen .

Frontiers of Medicine, 2015, 9 :90-99

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