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MXPA03008210A - Produccion de altos niveles del factor ix transgenico sin rescate del gen, y sus usos terapeuticos. - Google Patents

Produccion de altos niveles del factor ix transgenico sin rescate del gen, y sus usos terapeuticos.

Info

Publication number
MXPA03008210A
MXPA03008210A MXPA03008210A MXPA03008210A MXPA03008210A MX PA03008210 A MXPA03008210 A MX PA03008210A MX PA03008210 A MXPA03008210 A MX PA03008210A MX PA03008210 A MXPA03008210 A MX PA03008210A MX PA03008210 A MXPA03008210 A MX PA03008210A
Authority
MX
Mexico
Prior art keywords
animal
milk
gene
factor
trangenic
Prior art date
Application number
MXPA03008210A
Other languages
English (en)
Inventor
K O'sickey Tanya
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.)
Filing date
Publication date
Application filed by Progenetics Llc filed Critical Progenetics Llc
Publication of MXPA03008210A publication Critical patent/MXPA03008210A/es

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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
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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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
    • A01K67/0275Genetically modified vertebrates, e.g. transgenic
    • A01K67/0278Knock-in vertebrates, e.g. humanised vertebrates
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    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • 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
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702Regulators; Modulating activity
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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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    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • 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
    • C07K14/8107Endopeptidase (E.C. 3.4.21-99) inhibitors
    • C07K14/811Serine protease (E.C. 3.4.21) inhibitors
    • C07K14/8114Kunitz type inhibitors
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    • C12N9/14Hydrolases (3)
    • C12N9/48Hydrolases (3) acting on peptide bonds (3.4)
    • 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
    • C12N9/6421Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
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    • C12N9/64Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424Serine endopeptidases (3.4.21)
    • C12N9/6437Coagulation factor VIIa (3.4.21.21)
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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/48Hydrolases (3) acting on peptide bonds (3.4)
    • 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
    • C12N9/6421Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424Serine endopeptidases (3.4.21)
    • C12N9/644Coagulation factor IXa (3.4.21.22)
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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
    • C12N9/6421Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424Serine endopeptidases (3.4.21)
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    • C12Y304/21069Protein C activated (3.4.21.69)
    • 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
    • A01K2207/00Modified animals
    • A01K2207/15Humanized animals
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    • A01K2217/00Genetically modified animals
    • A01K2217/05Animals comprising random inserted nucleic acids (transgenic)
    • AHUMAN NECESSITIES
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    • A01K2227/10Mammal
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    • AHUMAN NECESSITIES
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    • C12N2830/00Vector systems having a special element relevant for transcription
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  • General Engineering & Computer Science (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
  • Veterinary Medicine (AREA)
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  • Animal Husbandry (AREA)
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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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Abstract

Un animal mamifero transgenico no humano, como es descrito anteriormente, contiene una secuencia de DNA de doble hebra, exogena, establemente integrada en el genoma del animal, que comprende unidades reguladoras de cis-accionamiento operablemente enlazadas a una secuencia de DNA que codifica la proteina FIX humana sin el beneficio de la presencia de una secuencia de gen de leche completa para el rescate del gen, y una secuencia de senal es activa en dirigir el Factor IX recien expresado en la leche del animal a niveles en una forma inactivada que es adecuada para el procesamiento subsecuente para aplicaciones terapeuticas en el tratamiento de la hemofilia B. Los mamiferos transgenicos de preferencia son cerdos, vacas, ovejas, cabras y conejos. La aplicacion incluye derivados de leche para el suministro oral y la tolerizacion oral en el tratamiento de la hemofilia B.
MXPA03008210A 2001-03-12 2002-03-11 Produccion de altos niveles del factor ix transgenico sin rescate del gen, y sus usos terapeuticos. MXPA03008210A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US27498301P 2001-03-12 2001-03-12
PCT/US2002/007532 WO2002077161A2 (en) 2001-03-12 2002-03-11 Production of high levels of trangenic factor ix without gene rescue, and its therapeutic uses

Publications (1)

Publication Number Publication Date
MXPA03008210A true MXPA03008210A (es) 2005-03-07

Family

ID=23050399

Family Applications (2)

Application Number Title Priority Date Filing Date
MXPA03008210A MXPA03008210A (es) 2001-03-12 2002-03-11 Produccion de altos niveles del factor ix transgenico sin rescate del gen, y sus usos terapeuticos.
MXPA03008209A MXPA03008209A (es) 2001-03-12 2002-03-11 Produccion de altos niveles del factor viii transgenico con estabilidad disenada, y sus usos terapeuticos.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MXPA03008209A MXPA03008209A (es) 2001-03-12 2002-03-11 Produccion de altos niveles del factor viii transgenico con estabilidad disenada, y sus usos terapeuticos.

Country Status (6)

Country Link
US (2) US7723563B2 (es)
EP (2) EP1402049A4 (es)
AU (1) AU2002252310A1 (es)
BR (2) BR0208029A (es)
MX (2) MXPA03008210A (es)
WO (3) WO2002072023A2 (es)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP4644663B2 (ja) 2003-06-03 2011-03-02 セル ジェネシス インコーポレイテッド ペプチド開裂部位を用いた単一ベクターからの組換えポリペプチドの高められた発現のための構成と方法
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
EP2097096B1 (en) 2006-12-22 2017-05-31 CSL Behring GmbH Modified coagulation factors with prolonged in vivo half-life
US20090235369A1 (en) 2007-12-31 2009-09-17 Baxter Healthcare S.A. Transgenic Non-Human Animals Expressing Human Blood Clotting Factors and Uses Thereof
AU2009266603B2 (en) * 2008-06-30 2013-01-24 Cho-A Pharm Co., Ltd. Gene of porcine alpha-s1 casein, a promoter of the same and use thereof
AU2008359017B2 (en) * 2008-06-30 2013-01-17 Cho-A Pharm Co., Ltd. A 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
US20240200093A1 (en) * 2021-04-09 2024-06-20 University Of Washington Artificial regulatory cassettes for muscle-specific gene expression

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
US5322775A (en) * 1986-06-30 1994-06-21 Pharmaceutical Proteins Ltd. 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
WO1994019935A1 (en) * 1993-03-09 1994-09-15 Genzyme Corporation Isolation of components of interest from milk
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

Also Published As

Publication number Publication date
US20040133932A1 (en) 2004-07-08
WO2002072024A2 (en) 2002-09-19
EP1402049A4 (en) 2004-09-01
BR0208029A (pt) 2004-07-27
WO2002077161A3 (en) 2003-04-03
MXPA03008209A (es) 2004-11-12
WO2002072023A2 (en) 2002-09-19
EP1402049A2 (en) 2004-03-31
AU2002252310A1 (en) 2002-09-24
US7723563B2 (en) 2010-05-25
EP1379677A4 (en) 2004-09-08
WO2002077161A2 (en) 2002-10-03
WO2002072024A3 (en) 2003-03-20
BR0208030A (pt) 2004-02-25
EP1379677A2 (en) 2004-01-14
US20100333218A1 (en) 2010-12-30
WO2002072023A3 (en) 2004-01-15

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