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SK-HEP cells and lentiviral vector for production of human recombinant factor VIII
Hemophilia A is caused by a deficiency in coagulation factor VIII. Recombinant factor VIII can be used as an alternative although it is unavailable for most patients. Here, we describe the production of a human recombinant B-domain-deleted FVIII (rBDDFVIII) by the human cell line SK-HEP-1, modified...
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| Published in: | Biotechnology letters 2012-08, Vol.34 (8), p.1435-1443 |
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| container_end_page | 1443 |
| container_issue | 8 |
| container_start_page | 1435 |
| container_title | Biotechnology letters |
| container_volume | 34 |
| creator | da Rosa, Nathalia Gonsales Swiech, Kamilla Picanço-Castro, Virgínia de Sousa Russo-Carbolante, Elisa Maria Neto, Mario Abreu Soares de Castilho-Fernandes, Andrielle Faça, Vitor Marcel Fontes, Aparecida Maria Covas, Dimas Tadeu |
| description | Hemophilia A is caused by a deficiency in coagulation factor VIII. Recombinant factor VIII can be used as an alternative although it is unavailable for most patients. Here, we describe the production of a human recombinant B-domain-deleted FVIII (rBDDFVIII) by the human cell line SK-HEP-1, modified by a lentiviral vector rBDDFVIII was produced by recombinant SK-HEP cells (rSK-HEP) at 1.5–2.1 IU/106 in 24 h. The recombinant factor had increased in vitro stability when compared to commercial pdFVIII. The functionality of rBDDFVIII was shown by its biological activity and by tail-clip challenge in hemophilia A mice. The rSK-HEP cells grew in a scalable system and produced active rBDDFVIII, indicating that this platform production can be optimized to meet the commercial production scale needs. |
| doi_str_mv | 10.1007/s10529-012-0925-4 |
| format | article |
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Recombinant factor VIII can be used as an alternative although it is unavailable for most patients. Here, we describe the production of a human recombinant B-domain-deleted FVIII (rBDDFVIII) by the human cell line SK-HEP-1, modified by a lentiviral vector rBDDFVIII was produced by recombinant SK-HEP cells (rSK-HEP) at 1.5–2.1 IU/106 in 24 h. The recombinant factor had increased in vitro stability when compared to commercial pdFVIII. The functionality of rBDDFVIII was shown by its biological activity and by tail-clip challenge in hemophilia A mice. The rSK-HEP cells grew in a scalable system and produced active rBDDFVIII, indicating that this platform production can be optimized to meet the commercial production scale needs.</description><identifier>ISSN: 0141-5492</identifier><identifier>EISSN: 1573-6776</identifier><identifier>DOI: 10.1007/s10529-012-0925-4</identifier><identifier>PMID: 22488441</identifier><identifier>CODEN: BILED3</identifier><language>eng</language><publisher>Dordrecht: Springer-Verlag</publisher><subject>Animals ; Applied Microbiology ; bioactive properties ; Biochemistry ; Biological and medical sciences ; Biomedical and Life Sciences ; Biotechnology ; Blotting, Western ; Cell Culture Techniques ; Cell Line ; Cells ; Coagulation ; Disease Models, Animal ; factor VIII ; Factor VIII - biosynthesis ; Factor VIII - chemistry ; Factor VIII - genetics ; Factor VIII - pharmacology ; Flow Cytometry ; Fundamental and applied biological sciences. Psychology ; Genetic Vectors - genetics ; Hemophilia ; Hemophilia A - drug therapy ; Human ; Humans ; Lentivirus - genetics ; Life Sciences ; Mathematical analysis ; Mice ; Microbiology ; Original Research Paper ; patients ; Peptide Fragments - biosynthesis ; Peptide Fragments - chemistry ; Peptide Fragments - genetics ; Peptide Fragments - pharmacology ; Platforms ; Proteins ; Recombinant ; Recombinant Proteins - biosynthesis ; Recombinant Proteins - chemistry ; Recombinant Proteins - genetics ; Recombinant Proteins - pharmacology ; Stability ; Survival Analysis ; Vectors (mathematics)</subject><ispartof>Biotechnology letters, 2012-08, Vol.34 (8), p.1435-1443</ispartof><rights>Springer Science+Business Media B.V. 2012</rights><rights>2015 INIST-CNRS</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c516t-a67846020e912af36a9ccfe55205c52ef905e37b45d915cf98089aaee1eca84f3</citedby><cites>FETCH-LOGICAL-c516t-a67846020e912af36a9ccfe55205c52ef905e37b45d915cf98089aaee1eca84f3</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><link.rule.ids>314,780,784,27924,27925</link.rule.ids><backlink>$$Uhttp://pascal-francis.inist.fr/vibad/index.php?action=getRecordDetail&idt=26193010$$DView record in Pascal Francis$$Hfree_for_read</backlink><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/22488441$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>da Rosa, Nathalia Gonsales</creatorcontrib><creatorcontrib>Swiech, Kamilla</creatorcontrib><creatorcontrib>Picanço-Castro, Virgínia</creatorcontrib><creatorcontrib>de Sousa Russo-Carbolante, Elisa Maria</creatorcontrib><creatorcontrib>Neto, Mario Abreu Soares</creatorcontrib><creatorcontrib>de Castilho-Fernandes, Andrielle</creatorcontrib><creatorcontrib>Faça, Vitor Marcel</creatorcontrib><creatorcontrib>Fontes, Aparecida Maria</creatorcontrib><creatorcontrib>Covas, Dimas Tadeu</creatorcontrib><title>SK-HEP cells and lentiviral vector for production of human recombinant factor VIII</title><title>Biotechnology letters</title><addtitle>Biotechnol Lett</addtitle><addtitle>Biotechnol Lett</addtitle><description>Hemophilia A is caused by a deficiency in coagulation factor VIII. Recombinant factor VIII can be used as an alternative although it is unavailable for most patients. Here, we describe the production of a human recombinant B-domain-deleted FVIII (rBDDFVIII) by the human cell line SK-HEP-1, modified by a lentiviral vector rBDDFVIII was produced by recombinant SK-HEP cells (rSK-HEP) at 1.5–2.1 IU/106 in 24 h. The recombinant factor had increased in vitro stability when compared to commercial pdFVIII. The functionality of rBDDFVIII was shown by its biological activity and by tail-clip challenge in hemophilia A mice. The rSK-HEP cells grew in a scalable system and produced active rBDDFVIII, indicating that this platform production can be optimized to meet the commercial production scale needs.</description><subject>Animals</subject><subject>Applied Microbiology</subject><subject>bioactive properties</subject><subject>Biochemistry</subject><subject>Biological and medical sciences</subject><subject>Biomedical and Life Sciences</subject><subject>Biotechnology</subject><subject>Blotting, Western</subject><subject>Cell Culture Techniques</subject><subject>Cell Line</subject><subject>Cells</subject><subject>Coagulation</subject><subject>Disease Models, Animal</subject><subject>factor VIII</subject><subject>Factor VIII - biosynthesis</subject><subject>Factor VIII - chemistry</subject><subject>Factor VIII - genetics</subject><subject>Factor VIII - pharmacology</subject><subject>Flow Cytometry</subject><subject>Fundamental and applied biological sciences. Psychology</subject><subject>Genetic Vectors - genetics</subject><subject>Hemophilia</subject><subject>Hemophilia A - drug therapy</subject><subject>Human</subject><subject>Humans</subject><subject>Lentivirus - genetics</subject><subject>Life Sciences</subject><subject>Mathematical analysis</subject><subject>Mice</subject><subject>Microbiology</subject><subject>Original Research Paper</subject><subject>patients</subject><subject>Peptide Fragments - biosynthesis</subject><subject>Peptide Fragments - chemistry</subject><subject>Peptide Fragments - genetics</subject><subject>Peptide Fragments - pharmacology</subject><subject>Platforms</subject><subject>Proteins</subject><subject>Recombinant</subject><subject>Recombinant Proteins - biosynthesis</subject><subject>Recombinant Proteins - chemistry</subject><subject>Recombinant Proteins - genetics</subject><subject>Recombinant Proteins - pharmacology</subject><subject>Stability</subject><subject>Survival Analysis</subject><subject>Vectors (mathematics)</subject><issn>0141-5492</issn><issn>1573-6776</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2012</creationdate><recordtype>article</recordtype><recordid>eNqNks1rFTEUxYMo9vn0D3CjAyK4id58TZKllGofFirWuh3uy0vqlJlMTWYK_vdmnGcrBT8WIYv87sk5nEvIUwavGYB-kxkobikwTsFyReU9smJKC1prXd8nK2CSUSUtPyCPcr4EAKtBPyQHnEtjpGQr8unsAz0--lg533W5wrirOh_H9rpN2FXX3o1DqkI5V2nYTW5sh1gNofo69Rir5N3Qb9uIcawC_kS_bDabx-RBwC77J_t7Tc7fHX0-PKYnp-83h29PqFOsHinW2sgaOHjLOAZRo3UueKU4KKe4DxaUF3or1c4y5YI1YCyi98w7NDKINXm16BZv3yafx6Zv85wDox-m3LDaKMMZF_rfKAhpmNDS_AfKTTFdTBb0xR30cphSLJlnSlvBhZoF2UK5NOScfGiuUttj-l6gZq6xWWpsSo3NXGMjy8yzvfK07f3uZuJXbwV4uQcwO-xCwujafMvVzAooqdaEL1wuT_HCp98t_vn358tQwKHBi1SEz894WaayQFJJ0H8luGa1Fj8AjiTDuw</recordid><startdate>20120801</startdate><enddate>20120801</enddate><creator>da Rosa, Nathalia Gonsales</creator><creator>Swiech, Kamilla</creator><creator>Picanço-Castro, Virgínia</creator><creator>de Sousa Russo-Carbolante, Elisa Maria</creator><creator>Neto, Mario Abreu Soares</creator><creator>de Castilho-Fernandes, Andrielle</creator><creator>Faça, Vitor Marcel</creator><creator>Fontes, Aparecida Maria</creator><creator>Covas, Dimas Tadeu</creator><general>Springer-Verlag</general><general>Springer Netherlands</general><general>Springer</general><general>Springer Nature B.V</general><scope>FBQ</scope><scope>IQODW</scope><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>3V.</scope><scope>7QL</scope><scope>7QR</scope><scope>7T7</scope><scope>7TB</scope><scope>7U5</scope><scope>7X7</scope><scope>7XB</scope><scope>88A</scope><scope>88E</scope><scope>88I</scope><scope>8AO</scope><scope>8FD</scope><scope>8FE</scope><scope>8FG</scope><scope>8FH</scope><scope>8FI</scope><scope>8FJ</scope><scope>8FK</scope><scope>ABJCF</scope><scope>ABUWG</scope><scope>AFKRA</scope><scope>AZQEC</scope><scope>BBNVY</scope><scope>BENPR</scope><scope>BGLVJ</scope><scope>BHPHI</scope><scope>C1K</scope><scope>CCPQU</scope><scope>DWQXO</scope><scope>FR3</scope><scope>FYUFA</scope><scope>GHDGH</scope><scope>GNUQQ</scope><scope>HCIFZ</scope><scope>K9.</scope><scope>L6V</scope><scope>L7M</scope><scope>LK8</scope><scope>M0S</scope><scope>M1P</scope><scope>M2P</scope><scope>M7N</scope><scope>M7P</scope><scope>M7S</scope><scope>P64</scope><scope>PQEST</scope><scope>PQQKQ</scope><scope>PQUKI</scope><scope>PTHSS</scope><scope>Q9U</scope><scope>7X8</scope><scope>7QO</scope><scope>7U9</scope><scope>H94</scope></search><sort><creationdate>20120801</creationdate><title>SK-HEP cells and lentiviral vector for production of human recombinant factor VIII</title><author>da Rosa, Nathalia Gonsales ; Swiech, Kamilla ; Picanço-Castro, Virgínia ; de Sousa Russo-Carbolante, Elisa Maria ; Neto, Mario Abreu Soares ; de Castilho-Fernandes, Andrielle ; Faça, Vitor Marcel ; Fontes, Aparecida Maria ; Covas, Dimas Tadeu</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c516t-a67846020e912af36a9ccfe55205c52ef905e37b45d915cf98089aaee1eca84f3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2012</creationdate><topic>Animals</topic><topic>Applied Microbiology</topic><topic>bioactive properties</topic><topic>Biochemistry</topic><topic>Biological and medical sciences</topic><topic>Biomedical and Life Sciences</topic><topic>Biotechnology</topic><topic>Blotting, Western</topic><topic>Cell Culture Techniques</topic><topic>Cell Line</topic><topic>Cells</topic><topic>Coagulation</topic><topic>Disease Models, Animal</topic><topic>factor VIII</topic><topic>Factor VIII - biosynthesis</topic><topic>Factor VIII - chemistry</topic><topic>Factor VIII - genetics</topic><topic>Factor VIII - pharmacology</topic><topic>Flow Cytometry</topic><topic>Fundamental and applied biological sciences. Psychology</topic><topic>Genetic Vectors - genetics</topic><topic>Hemophilia</topic><topic>Hemophilia A - drug therapy</topic><topic>Human</topic><topic>Humans</topic><topic>Lentivirus - genetics</topic><topic>Life Sciences</topic><topic>Mathematical analysis</topic><topic>Mice</topic><topic>Microbiology</topic><topic>Original Research Paper</topic><topic>patients</topic><topic>Peptide Fragments - biosynthesis</topic><topic>Peptide Fragments - chemistry</topic><topic>Peptide Fragments - genetics</topic><topic>Peptide Fragments - pharmacology</topic><topic>Platforms</topic><topic>Proteins</topic><topic>Recombinant</topic><topic>Recombinant Proteins - biosynthesis</topic><topic>Recombinant Proteins - chemistry</topic><topic>Recombinant Proteins - genetics</topic><topic>Recombinant Proteins - pharmacology</topic><topic>Stability</topic><topic>Survival Analysis</topic><topic>Vectors (mathematics)</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>da Rosa, Nathalia Gonsales</creatorcontrib><creatorcontrib>Swiech, Kamilla</creatorcontrib><creatorcontrib>Picanço-Castro, Virgínia</creatorcontrib><creatorcontrib>de Sousa Russo-Carbolante, Elisa Maria</creatorcontrib><creatorcontrib>Neto, Mario Abreu Soares</creatorcontrib><creatorcontrib>de Castilho-Fernandes, Andrielle</creatorcontrib><creatorcontrib>Faça, Vitor Marcel</creatorcontrib><creatorcontrib>Fontes, Aparecida Maria</creatorcontrib><creatorcontrib>Covas, Dimas Tadeu</creatorcontrib><collection>AGRIS</collection><collection>Pascal-Francis</collection><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>ProQuest Central (Corporate)</collection><collection>Bacteriology Abstracts (Microbiology B)</collection><collection>Chemoreception Abstracts</collection><collection>Industrial and Applied Microbiology Abstracts (Microbiology A)</collection><collection>Mechanical & Transportation Engineering Abstracts</collection><collection>Solid State and Superconductivity Abstracts</collection><collection>Health & Medical Collection</collection><collection>ProQuest Central (purchase pre-March 2016)</collection><collection>Biology Database (Alumni Edition)</collection><collection>Medical Database (Alumni Edition)</collection><collection>Science Database (Alumni Edition)</collection><collection>ProQuest Pharma Collection</collection><collection>Technology Research Database</collection><collection>ProQuest SciTech Collection</collection><collection>ProQuest Technology Collection</collection><collection>ProQuest Natural Science Collection</collection><collection>Hospital Premium Collection</collection><collection>Hospital Premium Collection (Alumni Edition)</collection><collection>ProQuest Central (Alumni) (purchase pre-March 2016)</collection><collection>Materials Science & Engineering Collection</collection><collection>ProQuest Central (Alumni)</collection><collection>ProQuest Central</collection><collection>ProQuest Central Essentials</collection><collection>Biological Science Collection</collection><collection>ProQuest Central</collection><collection>Technology Collection</collection><collection>Natural Science Collection</collection><collection>Environmental Sciences and Pollution Management</collection><collection>ProQuest One Community College</collection><collection>ProQuest Central</collection><collection>Engineering Research Database</collection><collection>Health Research Premium Collection</collection><collection>Health Research Premium Collection (Alumni)</collection><collection>ProQuest Central Student</collection><collection>SciTech Premium Collection</collection><collection>ProQuest Health & Medical Complete (Alumni)</collection><collection>ProQuest Engineering Collection</collection><collection>Advanced Technologies Database with Aerospace</collection><collection>ProQuest Biological Science Collection</collection><collection>Health & Medical Collection (Alumni Edition)</collection><collection>PML(ProQuest Medical Library)</collection><collection>Science Database</collection><collection>Algology Mycology and Protozoology Abstracts (Microbiology C)</collection><collection>Biological Science Database</collection><collection>Engineering Database</collection><collection>Biotechnology and BioEngineering Abstracts</collection><collection>ProQuest One Academic Eastern Edition (DO NOT USE)</collection><collection>ProQuest One Academic</collection><collection>ProQuest One Academic UKI Edition</collection><collection>Engineering Collection</collection><collection>ProQuest Central Basic</collection><collection>MEDLINE - Academic</collection><collection>Biotechnology Research Abstracts</collection><collection>Virology and AIDS Abstracts</collection><collection>AIDS and Cancer Research Abstracts</collection><jtitle>Biotechnology letters</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>da Rosa, Nathalia Gonsales</au><au>Swiech, Kamilla</au><au>Picanço-Castro, Virgínia</au><au>de Sousa Russo-Carbolante, Elisa Maria</au><au>Neto, Mario Abreu Soares</au><au>de Castilho-Fernandes, Andrielle</au><au>Faça, Vitor Marcel</au><au>Fontes, Aparecida Maria</au><au>Covas, Dimas Tadeu</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>SK-HEP cells and lentiviral vector for production of human recombinant factor VIII</atitle><jtitle>Biotechnology letters</jtitle><stitle>Biotechnol Lett</stitle><addtitle>Biotechnol Lett</addtitle><date>2012-08-01</date><risdate>2012</risdate><volume>34</volume><issue>8</issue><spage>1435</spage><epage>1443</epage><pages>1435-1443</pages><issn>0141-5492</issn><eissn>1573-6776</eissn><coden>BILED3</coden><abstract>Hemophilia A is caused by a deficiency in coagulation factor VIII. Recombinant factor VIII can be used as an alternative although it is unavailable for most patients. Here, we describe the production of a human recombinant B-domain-deleted FVIII (rBDDFVIII) by the human cell line SK-HEP-1, modified by a lentiviral vector rBDDFVIII was produced by recombinant SK-HEP cells (rSK-HEP) at 1.5–2.1 IU/106 in 24 h. The recombinant factor had increased in vitro stability when compared to commercial pdFVIII. The functionality of rBDDFVIII was shown by its biological activity and by tail-clip challenge in hemophilia A mice. The rSK-HEP cells grew in a scalable system and produced active rBDDFVIII, indicating that this platform production can be optimized to meet the commercial production scale needs.</abstract><cop>Dordrecht</cop><pub>Springer-Verlag</pub><pmid>22488441</pmid><doi>10.1007/s10529-012-0925-4</doi><tpages>9</tpages></addata></record> |
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| subjects | Animals Applied Microbiology bioactive properties Biochemistry Biological and medical sciences Biomedical and Life Sciences Biotechnology Blotting, Western Cell Culture Techniques Cell Line Cells Coagulation Disease Models, Animal factor VIII Factor VIII - biosynthesis Factor VIII - chemistry Factor VIII - genetics Factor VIII - pharmacology Flow Cytometry Fundamental and applied biological sciences. Psychology Genetic Vectors - genetics Hemophilia Hemophilia A - drug therapy Human Humans Lentivirus - genetics Life Sciences Mathematical analysis Mice Microbiology Original Research Paper patients Peptide Fragments - biosynthesis Peptide Fragments - chemistry Peptide Fragments - genetics Peptide Fragments - pharmacology Platforms Proteins Recombinant Recombinant Proteins - biosynthesis Recombinant Proteins - chemistry Recombinant Proteins - genetics Recombinant Proteins - pharmacology Stability Survival Analysis Vectors (mathematics) |
| title | SK-HEP cells and lentiviral vector for production of human recombinant factor VIII |
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