Recombinant plasma proteins

Vox Sanguinis - Tập 100 Số 1 - Trang 68-83 - 2011
Thierry Burnouf1
1Human Protein Process Sciences (HPPS), Lille, France

Tóm tắt

For almost 50 years, the fractionation of human plasma has been the sole possible source of a wide range of therapeutic proteins–such as coagulation factors, anticoagulants, immunoglobulins, and albumin – essential to the treatment of serious congenital or acquired bleeding or immunological diseases. In the last 20 years, the application of recombinant technologies to mammalian cell cultures has made possible – although with some limitations in productivity, costs, and immunogenic risks – to produce and commercialize complex plasma glycoproteins for human therapeutic applications and has opened the way to the development of new molecular entities. Today, the advanced exploration of alternative cell lines and enhanced cell culture systems, as well as the development of alternative expression technologies, such as transgenic animals, is opening a new era in the production of the full range of recombinant plasma protein therapeutics. In this review, we examine the achievements and ongoing challenges of the recombinant DNA technology as a platform for the production of plasma proteins and the advantages and limitations of such products compared to fractionated plasma proteins.

Từ khóa


Tài liệu tham khảo

10.1021/ja01207a034

10.1016/j.tmrv.2006.11.001

10.1016/j.tracli.2007.04.002

10.1016/j.beha.2005.01.002

10.1038/312330a0

10.1073/pnas.79.21.6461

10.1160/TH07-10-0593

10.2741/3024

10.1016/j.biotechadv.2009.01.008

10.1186/1475-2859-8-17

10.1007/s10529-009-0077-3

10.1016/S0076-6879(09)63011-1

10.1007/s00726-005-0324-4

Weidner M, 2010, Expression of recombinant proteins in the methylotrophic yeast Pichia pastoris, J Vis Exp, 36

10.1186/1475-2859-8-51

10.1016/j.biologicals.2005.08.021

10.1016/j.tibtech.2005.06.002

10.1007/978-1-60761-232-2_9

10.2174/187220809787172669

10.1016/S0958-1669(00)00197-X

10.1111/j.1538-7836.2005.01367.x

10.1038/nbt1026

10.1002/biot.200800241

10.1023/A:1008934912555

10.1111/j.1365-2516.2004.00983.x

Echelard Y, 2006, Production of Recombinant Therapeutic Proteins in the Milk of Transgenic Animals, Biopharm Int, 19, 36

10.1071/RD07040

10.1016/S1387-2656(07)13006-4

10.1038/nbt1027

10.1016/j.tibtech.2008.05.007

10.1007/978-1-59745-407-0_9

10.1007/978-3-540-70868-1_4

Kaufman RJ, 1986, Expression, purification, and characterization of recombinant gamma‐carboxylated factor IX synthesized in Chinese hamster ovary cells, J Biol Chem, 261, 9622, 10.1016/S0021-9258(18)67559-3

10.1055/s-0038-1657536

10.1182/blood-2004-06-2283

White G, 1998, Clinical evaluation of recombinant factor IX, Semin Hematol, 35, 33

10.1007/s10529-008-9818-y

10.1042/BA20060224

10.1038/312326a0

10.1038/312337a0

10.1054/blre.2000.0129

Eaton DL, 1987, Characterization of recombinant human factor VIII, J Biol Chem, 262, 3285, 10.1016/S0021-9258(18)61502-9

10.1385/MB:34:2:165

10.1016/j.tracli.2006.11.001

10.1016/j.biologicals.2009.01.005

10.1111/j.1365-2516.2008.01936.x

10.1111/j.1365-2516.2009.02135.x

10.1182/blood-2009-11-254755

10.1023/B:PHAM.0000029275.41323.a6

10.1182/blood-2005-04-1371

10.1111/j.1538-7836.2010.03823.x

10.1182/blood-2008-09-174649

10.1111/j.1365-2516.2006.01418.x

10.1055/s-0037-1613191

10.1182/blood-2006-11-056317

10.1055/s-2000-9821

10.1016/j.jbiotec.2006.03.042

10.1046/j.1423-0410.2003.00247.x

10.1111/j.1423-0410.2006.00888.x

10.1182/blood-2008-10-165621

10.1111/j.1365-2516.2008.01847.x

10.1016/j.biologicals.2005.11.010

10.1111/j.1423-0410.1992.tb01159.x

Turecek PL, 2009, Development of a plasma‐ and albumin‐free recombinant von Willebrand factor, Hamostaseologie, 29, S32

10.1055/s-2001-16892

10.1262/jrd.20212

10.1111/j.1749-6632.2001.tb03491.x

Townsend RR, 1982, Carbohydrate structure of human fibrinogen. Use of 300‐MHz 1H‐NMR to characterize glycosidase‐treated glycopeptides, J Biol Chem, 257, 9704, 10.1016/S0021-9258(18)34130-9

10.1097/00001721-199611000-00001

10.1046/j.1423-0410.1997.7230133.x

Mintz PD, 2001, Fibrin sealant: clinical use and the development of the University of Virginia Tissue Adhesive Center, Ann Clin Lab Sci, 31, 108

10.1016/j.pep.2008.02.010

10.1007/s11248-004-9589-8

Butler SP, 1997, Current progress in the production of recombinant human fibrinogen in the milk of transgenic animals, Thromb Haemost, 78, 537, 10.1055/s-0038-1657584

10.1161/01.ATV.0000046238.23903.FC

10.1038/nbt0308-250

10.1016/j.thromres.2007.12.027

10.1016/j.clinthera.2009.01.005

10.1185/03007990802186417

10.1038/39057

Semple E, 2005, Quality of thrombin produced from the patient’s own plasma using the TPD, a new Thrombin‐processing Device, J Extra Corpor Technol, 37, 196, 10.1051/ject/200537196

10.1055/s-2006-939558

10.1517/14712590802588773

10.1016/j.jamcollsurg.2009.09.042

10.1080/21548331.1991.11704145

10.1517/14656560903300111

10.1001/archderm.142.3.396

10.1056/NEJM200505123521923

10.1097/00001721-200212000-00001

Rodgers GM, 2009, Role of antithrombin concentrate in treatment of hereditary antithrombin deficiency An update, Thromb Haemost, 101, 806, 10.1160/TH08-10-0672

10.2165/00003495-200767100-00005

10.1002/bit.21758

10.1177/1076029609339748

Berry LR, 2009, Comparison of recombinant and plasma‐derived antithrombin biodistribution in a rabbit model, Thromb Haemost, 102, 302, 10.1160/TH09-01-0062

Caliezi C, 2000, C1‐Esterase inhibitor: an anti‐inflammatory agent and its potential use in the treatment of diseases other than hereditary angioedema, Pharmacol Rev, 52, 91

10.1111/j.1365-2249.2005.02726.x

10.1016/j.molmed.2008.12.001

10.1111/j.1423-0410.2007.00953.x

10.1111/j.1423-0410.1995.tb00366.x

10.1056/NEJM200103083441001

10.1006/biol.1998.0163

WHO, 2003, Guidelines on Viral Inactivation and Removal Procedures Intended to Assure the Viral Safety of Human Blood Plasma Products

10.1358/dot.2007.43.8.1067343

10.1016/S1389-1723(00)88050-0

10.1111/j.1744-9987.1998.tb00118.x

10.1023/A:1008081314112

10.1006/abio.1997.2480

Dodsworth N, 1996, Comparative studies of recombinant human albumin and human serum albumin derived by blood fractionation, Biotechnol Appl Biochem, 24, 171, 10.1111/j.1470-8744.1996.tb00397.x

10.1177/0091270004269646

10.1007/s00535-008-2178-5

10.1177/0091270007310549

10.1007/s11248-008-9229-9

10.1016/j.jaci.2009.08.018

10.1007/s10875-010-9394-5

10.1385/CRIAI:29:3:167

10.1007/s12016-009-8155-9

10.1038/nrd2358-c1

10.1038/ncprheum0481

10.1146/annurev.immunol.26.021607.090232

10.1146/annurev.med.58.061705.145053

10.2165/11533070-000000000-00000

10.1111/j.1423-0410.2009.01226.x

10.1038/nrd3003

10.1111/j.1476-5381.2009.00190.x

10.1517/14712591003724670

10.2174/092986710790416245

10.1056/NEJMoa063842

10.3324/haematol.2008.001628

10.1016/j.vaccine.2009.09.105

10.1038/nrd1987

10.1371/journal.pntd.0000542

10.1016/S1474-4422(10)70043-0

10.1097/01.cji.0000192105.24583.83

10.1073/pnas.0914503107

10.1016/j.copbio.2009.10.008

Tuddenham EGD, 2010, From human to humans – introducing the first recombinant human FVIII product produced from a human cell line, Thromb Haemost, 103, 4

10.1111/j.1537-2995.2007.01239.x

10.1007/978-3-540-73259-4_1