BR0208029A - Produção de altos nìveis do fator transgênico viii com estabilidade elaborada e seus usos terapêuticos - Google Patents

Produção de altos nìveis do fator transgênico viii com estabilidade elaborada e seus usos terapêuticos

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Publication number
BR0208029A
BR0208029A BR0208029-0A BR0208029A BR0208029A BR 0208029 A BR0208029 A BR 0208029A BR 0208029 A BR0208029 A BR 0208029A BR 0208029 A BR0208029 A BR 0208029A
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BR
Brazil
Prior art keywords
factor viii
animal
transgenic
production
high levels
Prior art date
Application number
BR0208029-0A
Other languages
English (en)
Inventor
Julian D Cooper
William Hugold Velander
Stephen P Butler
Original Assignee
Progenetics Llc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
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Publication date
Application filed by Progenetics Llc filed Critical Progenetics Llc
Publication of BR0208029A publication Critical patent/BR0208029A/pt

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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/8509Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027New or modified breeds of vertebrates
    • A01K67/0275Genetically modified vertebrates, e.g. transgenic
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    • A01K67/00Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027New or modified breeds of vertebrates
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    • A01K67/0278Knock-in vertebrates, e.g. humanised vertebrates
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    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/72Receptors; Cell surface antigens; Cell surface determinants for hormones
    • C07K14/721Steroid/thyroid hormone superfamily, e.g. GR, EcR, androgen receptor, oestrogen receptor
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    • C07K14/745Blood coagulation or fibrinolysis factors
    • C07K14/755Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
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    • C07K14/81Protease inhibitors
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    • C12N9/64Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
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    • C12N9/64Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
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    • C12N9/14Hydrolases (3)
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    • C12N9/50Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
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    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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  • Health & Medical Sciences (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
  • Veterinary Medicine (AREA)
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Abstract

"PRODUçãO DE ALTOS NìVEIS DO FATOR TRANSGêNICO VIII COM ESTABILIDADE ELABORADA E SEUS USOS TERAPêUTICOS". Um animal mamífero transgênico não humano, conforme descrito acima, contém uma seq³ência de DNA de dupla fita exógena estavelmente integrada ao genoma do animal, que compreende unidades regulatórias cis-atuantes operacionalmente ligadas a uma seq³ência de DNA que codifica uma proteína FVIII humana variante ou modificada, e uma seq³ência sinal e fragmentos de secreção que sejam ativos no direcionamento do Fator VIII recentemente expressada no leite do animal em níveis e para uma forma e não ativada, não degradada e de outra forma estável que seja adequada para o processamento subseq³ente para aplicações terapêuticas no tratamento da Hemofilia A. Os mamiferos transgênicos são preferencialmente porcos, vacas, ovelhas, cabras e coelhos. As aplicações incluem derivados do leite utilizados para fornecimento oral e tolerância oral no tratamento da Hemofilia A.
BR0208029-0A 2001-03-12 2002-03-11 Produção de altos nìveis do fator transgênico viii com estabilidade elaborada e seus usos terapêuticos BR0208029A (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US27498301P 2001-03-12 2001-03-12
PCT/US2002/007530 WO2002072023A2 (en) 2001-03-12 2002-03-11 Production of high levels of transgenic factor viii with engineered stability, and its therapeutic uses

Publications (1)

Publication Number Publication Date
BR0208029A true BR0208029A (pt) 2004-07-27

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ID=23050399

Family Applications (2)

Application Number Title Priority Date Filing Date
BR0208030-3A BR0208030A (pt) 2001-03-12 2002-03-11 Produção de altos nìveis de fator ix transgênico sem resgate do gene e seus usos terapêuticos
BR0208029-0A BR0208029A (pt) 2001-03-12 2002-03-11 Produção de altos nìveis do fator transgênico viii com estabilidade elaborada e seus usos terapêuticos

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BR0208030-3A BR0208030A (pt) 2001-03-12 2002-03-11 Produção de altos nìveis de fator ix transgênico sem resgate do gene e seus usos terapêuticos

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US (2) US7723563B2 (pt)
EP (2) EP1379677A4 (pt)
AU (1) AU2002252310A1 (pt)
BR (2) BR0208030A (pt)
MX (2) MXPA03008210A (pt)
WO (3) WO2002077161A2 (pt)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005017149A1 (en) 2003-06-03 2005-02-24 Cell Genesys, Inc. Compositions and methods for enhanced expression of recombinant polypeptides from a single vector using a peptide cleavage site
US7485291B2 (en) 2003-06-03 2009-02-03 Cell Genesys, Inc. Compositions and methods for generating multiple polypeptides from a single vector using a virus derived peptide cleavage site, and uses thereof
EP1816201A1 (en) 2006-02-06 2007-08-08 CSL Behring GmbH Modified coagulation factor VIIa with extended half-life
FR2901796A1 (fr) * 2006-05-31 2007-12-07 Lab Francais Du Fractionnement Procede d'extraction d'une ou de plusieurs proteines presentes dans du lait
AU2007338298B2 (en) 2006-12-22 2013-02-07 Csl Behring Gmbh Modified coagulation factors with prolonged in vivo half-life
AR070089A1 (es) 2007-12-31 2010-03-10 Baxter Int Animales no humanos transgenicos que expresan factores de la coagula-cion sanguinea humanos y usos de los mismos
US9738694B2 (en) * 2008-06-30 2017-08-22 Cho-A Pharm. Co., Ltd. Gene of porcine alpha-s1 casein, a promoter of the same and use thereof
US8420388B2 (en) * 2008-06-30 2013-04-16 Cho-A Pharm. Co., Ltd. Gene of porcine beta casein, a promoter of the same and the use thereof
US20140115728A1 (en) 2012-10-24 2014-04-24 A. Joseph Tector Double knockout (gt/cmah-ko) pigs, organs and tissues
FR3011250A1 (fr) 2013-09-30 2015-04-03 Lab Francais Du Fractionnement Acides nucleiques se liant specifiquement au facteur ix/ixa humain, et utilisations
JP2024513907A (ja) * 2021-04-09 2024-03-27 ユニバーシティ オブ ワシントン 筋特異的遺伝子発現用の人工調節カセット

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4348384A (en) * 1980-10-17 1982-09-07 Dainippon Pharmaceutical Co., Ltd. Pharmaceutical composition for oral administration containing coagulation factor VIII or IX
US5366894A (en) * 1986-06-30 1994-11-22 Pharmaceutical Proteins Limited Peptide production
US5831141A (en) 1991-01-11 1998-11-03 United States Of America As Represented By The Department Of Health And Human Services Expression of a heterologous polypeptide in mammary tissue of transgenic non-human mammals using a long whey acidic protein promoter
DK0804070T3 (da) * 1993-03-09 2000-08-07 Genzyme Corp Fremgangsmåde til isolering af proteiner fra mælk
GB9408717D0 (en) * 1994-05-03 1994-06-22 Biotech & Biolog Scien Res DNA sequences
US5880327A (en) * 1994-09-21 1999-03-09 American National Red Cross Transgenic mammals expressing human coagulation factor VIII
DE69841459D1 (en) * 1997-02-14 2010-03-11 American Nat Red Cross Expression des active human factor ix im brustdrüsengewebe transgener tiere
US6087166A (en) 1997-07-03 2000-07-11 Basf Aktiengesellschaft Transcriptional activators with graded transactivation potential

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WO2002077161A3 (en) 2003-04-03
BR0208030A (pt) 2004-02-25
AU2002252310A1 (en) 2002-09-24
US7723563B2 (en) 2010-05-25
WO2002072023A3 (en) 2004-01-15
EP1402049A2 (en) 2004-03-31
US20040133932A1 (en) 2004-07-08
WO2002072023A2 (en) 2002-09-19
EP1379677A4 (en) 2004-09-08
MXPA03008209A (es) 2004-11-12
WO2002072024A3 (en) 2003-03-20
WO2002072024A2 (en) 2002-09-19
US20100333218A1 (en) 2010-12-30
EP1379677A2 (en) 2004-01-14
MXPA03008210A (es) 2005-03-07
EP1402049A4 (en) 2004-09-01
WO2002077161A2 (en) 2002-10-03

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