DK0845939T3 - Lysosomale proteiner produceret i mælk fra ikke-humane transgene dyr - Google Patents

Lysosomale proteiner produceret i mælk fra ikke-humane transgene dyr

Info

Publication number
DK0845939T3
DK0845939T3 DK96928405T DK96928405T DK0845939T3 DK 0845939 T3 DK0845939 T3 DK 0845939T3 DK 96928405 T DK96928405 T DK 96928405T DK 96928405 T DK96928405 T DK 96928405T DK 0845939 T3 DK0845939 T3 DK 0845939T3
Authority
DK
Denmark
Prior art keywords
milk
lysosomal proteins
transgenic animals
human transgenic
proteins produced
Prior art date
Application number
DK96928405T
Other languages
English (en)
Inventor
Arnold J J Reuser
Der Ploeg Ans T Van
Frank R Pieper
Martin Ph Verbeet
Original Assignee
Genzyme Corp
Univ Erasmus
Academic Hospital Rotterdam
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=21697877&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=DK0845939(T3) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Genzyme Corp, Univ Erasmus, Academic Hospital Rotterdam filed Critical Genzyme Corp
Application granted granted Critical
Publication of DK0845939T3 publication Critical patent/DK0845939T3/da

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Classifications

    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0276—Knock-out vertebrates
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0278—Knock-in vertebrates, e.g. humanised vertebrates
    • A—HUMAN NECESSITIES
    • A23—FOODS OR FOODSTUFFS; TREATMENT THEREOF, NOT COVERED BY OTHER CLASSES
    • A23C—DAIRY PRODUCTS, e.g. MILK, BUTTER OR CHEESE; MILK OR CHEESE SUBSTITUTES; MAKING THEREOF
    • A23C9/00—Milk preparations; Milk powder or milk powder preparations
    • A23C9/20—Dietetic milk products not covered by groups A23C9/12 - A23C9/18
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4732—Casein
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2405—Glucanases
    • C12N9/2408—Glucanases acting on alpha -1,4-glucosidic bonds
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2207/00—Modified animals
    • A01K2207/15—Humanized animals
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/107—Rabbit
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/01—Animal expressing industrially exogenous proteins
    • A—HUMAN NECESSITIES
    • A23—FOODS OR FOODSTUFFS; TREATMENT THEREOF, NOT COVERED BY OTHER CLASSES
    • A23C—DAIRY PRODUCTS, e.g. MILK, BUTTER OR CHEESE; MILK OR CHEESE SUBSTITUTES; MAKING THEREOF
    • A23C2230/00—Aspects relating to animal feed or genotype
    • A23C2230/05—Milk or milk products from transgenic animals
    • A—HUMAN NECESSITIES
    • A23—FOODS OR FOODSTUFFS; TREATMENT THEREOF, NOT COVERED BY OTHER CLASSES
    • A23C—DAIRY PRODUCTS, e.g. MILK, BUTTER OR CHEESE; MILK OR CHEESE SUBSTITUTES; MAKING THEREOF
    • A23C2230/00—Aspects relating to animal feed or genotype
    • A23C2230/15—Animal milk with modified composition due to manipulation of the animal, e.g. animal milk comprising antibodies, selection of animals having specific genotypes
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/85—Vector systems having a special element relevant for transcription from vertebrates mammalian
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y10—TECHNICAL SUBJECTS COVERED BY FORMER USPC
    • Y10S—TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y10S119/00—Animal husbandry
    • Y10S119/01—Milk digest

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Environmental Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Animal Husbandry (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Toxicology (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Nutrition Science (AREA)
  • Food Science & Technology (AREA)
  • Polymers & Plastics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Feed For Specific Animals (AREA)
DK96928405T 1995-08-02 1996-07-31 Lysosomale proteiner produceret i mælk fra ikke-humane transgene dyr DK0845939T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US179695P 1995-08-02 1995-08-02
PCT/EP1996/003421 WO1997005771A2 (en) 1995-08-02 1996-07-31 Lysosomal proteins produced in the milk of transgenic animals

Publications (1)

Publication Number Publication Date
DK0845939T3 true DK0845939T3 (da) 2003-09-22

Family

ID=21697877

Family Applications (1)

Application Number Title Priority Date Filing Date
DK96928405T DK0845939T3 (da) 1995-08-02 1996-07-31 Lysosomale proteiner produceret i mælk fra ikke-humane transgene dyr

Country Status (11)

Country Link
US (5) US6118045A (da)
EP (1) EP0845939B1 (da)
JP (1) JP2000501602A (da)
KR (1) KR19990036084A (da)
AT (1) ATE240637T1 (da)
AU (1) AU734953B2 (da)
DE (1) DE69628306T2 (da)
DK (1) DK0845939T3 (da)
ES (1) ES2200071T3 (da)
PT (1) PT845939E (da)
WO (1) WO1997005771A2 (da)

Families Citing this family (60)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040161837A1 (en) * 1995-08-02 2004-08-19 Reuser Arnold J. Methods of purifying human acid alpha-glucosidase
US20020013953A1 (en) * 1995-08-02 2002-01-31 Reuser Arnold J. Compositions and methods for treating enzyme deficiency
US6118045A (en) * 1995-08-02 2000-09-12 Pharming B.V. Lysosomal proteins produced in the milk of transgenic animals
WO1998037224A1 (en) * 1997-02-25 1998-08-27 Genzyme Transgenics Corporation Transgenically produced non-secreted proteins
EP1793000A1 (en) * 1997-02-25 2007-06-06 GTC Biotherapeutics, Inc. Transgenically produced non-secreted proteins
CA2353522A1 (en) * 1998-12-07 2000-06-15 Pharming Intellectual Property B.V. Treatment of pompe's disease
EP1958503A3 (en) * 1999-01-06 2008-11-26 Merrimack Pharmaceuticals, Inc. Expression of secreted human alpha-fetoprotein in transgenic animals
US7361481B2 (en) * 1999-04-09 2008-04-22 Women's And Children's Hospital Diagnosis of lysosomal storage disorders using saposins and other markers
US6537785B1 (en) 1999-09-14 2003-03-25 Genzyme Glycobiology Research Institute, Inc. Methods of treating lysosomal storage diseases
US6770468B1 (en) 1999-09-14 2004-08-03 Genzyme Glycobiology Research Institute, Inc. Phosphodiester-α-GlcNAcase of the lysosomal targeting pathway
ES2328446T5 (es) 2000-02-04 2014-02-27 Children's Hospital Research Foundation Uso de lipasa ácida lisosomal para tratar la aterosclerosis y enfermedades asociadas
PT3449934T (pt) 2000-07-18 2020-06-25 Univ Duke Tratamento de doença de armazenanto de glicogénio tipo ii
US7723296B2 (en) 2001-01-18 2010-05-25 Genzyme Corporation Methods for introducing mannose-6-phosphate and other oligosaccharides onto glycoproteins and its application thereof
US7001994B2 (en) * 2001-01-18 2006-02-21 Genzyme Corporation Methods for introducing mannose 6-phosphate and other oligosaccharides onto glycoproteins
EP1974752B1 (en) * 2001-04-30 2012-09-26 BioMarin Pharmaceutical Inc. Subcellular targeting of therapeutic proteins
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US20040005309A1 (en) * 2002-05-29 2004-01-08 Symbiontics, Inc. Targeted therapeutic proteins
US7629309B2 (en) * 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US20030072761A1 (en) * 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
US6800472B2 (en) 2001-12-21 2004-10-05 Genzyme Glycobiology Research Institute, Inc. Expression of lysosomal hydrolase in cells expressing pro-N-acetylglucosamine-1-phosphodiester α-N-acetyl glucosimanidase
US20040210953A1 (en) * 2003-01-09 2004-10-21 Murray James D. Lysozyme transgenic ungulates
US20040231010A1 (en) * 2003-01-09 2004-11-18 Murray James D. Lysozyme transgenic ungulates
WO2004064750A2 (en) * 2003-01-22 2004-08-05 Duke University Improved constructs for expressing lysosomal polypeptides
AU2004268207A1 (en) * 2003-08-21 2005-03-10 Q Therapeutics, Inc. Genetically modified somatic cells for sustained secretion of lysosomal proenzymes deficient in lysosomal storage disorders
JP4914224B2 (ja) * 2004-02-10 2012-04-11 バイオマリン ファーマシューティカル インコーポレイテッド 酸性αグルコシダーゼおよびそのフラグメント
US7740861B2 (en) * 2004-06-16 2010-06-22 University Of Massachusetts Drug delivery product and methods
US8889127B2 (en) * 2004-07-01 2014-11-18 Icahn School Of Medicine At Mount Sinai Targeted protein replacement for the treatment of lysosomal storage disorders
ATE536175T1 (de) 2004-09-17 2011-12-15 Univ Massachusetts Zusammensetzungen und ihre verwendungen bei lysosomalen enzymstörungen
US9181184B2 (en) 2005-05-17 2015-11-10 Amicus Therapeutics, Inc. Method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
US20090209624A1 (en) * 2005-10-24 2009-08-20 University Of Massachusetts Compositions and their uses for gene therapy of bone conditions
CA2669347A1 (en) * 2006-11-13 2008-05-29 Zystor Therapeutics, Inc. Methods for treating pompe disease
LT2457920T (lt) 2007-01-18 2018-02-12 Genzyme Corporation Oligosacharidai, apimantys aminooksigrupę, ir jų konjugatai
WO2009058913A2 (en) * 2007-10-29 2009-05-07 University Of Massachusetts Encapsulated nanoparticles for nucleic acid delivery
AU2009244148B2 (en) 2008-05-07 2014-10-09 Biomarin Pharmaceutical Inc. Lysosomal targeting peptides and uses thereof
JP5658167B2 (ja) 2008-12-16 2015-01-21 ジェンザイム・コーポレーション オリゴ糖−タンパク複合体
EP3075386B1 (en) 2009-06-17 2019-10-16 BioMarin Pharmaceutical Inc. Formulations for lysosomal enzymes
EP2561069B1 (en) 2010-04-23 2017-03-08 Alexion Pharmaceuticals, Inc. Lysosomal storage disease enzyme
KR20140005842A (ko) 2010-06-25 2014-01-15 샤이어 휴먼 지네틱 테라피즈 인크. 헤파란 n-설파타제의 cns 전달을 위한 방법들 및 조성물들
UA115649C2 (uk) 2010-06-25 2017-12-11 Шае Хюмен Дженетік Терапіс, Інк. Способи та композиції для доставки до цнс ідуронат-2-сульфатази
EP2585105B1 (en) 2010-06-25 2017-08-09 Shire Human Genetic Therapies, Inc. Treatment of sanfilippo syndrome type b
ME03647B (me) 2010-06-25 2020-07-20 Shire Human Genetic Therapies Postupci i kompozicije za isporuku arilsulfataze a u cns
PT2585104T (pt) 2010-06-25 2019-10-30 Shire Human Genetic Therapies Métodos e composições de arilsulfatase a para a administração no snc
KR102283671B1 (ko) 2010-06-25 2021-07-30 샤이어 휴먼 지네틱 테라피즈 인크. 치료제들의 cns 전달
KR20150038636A (ko) 2010-09-09 2015-04-08 시나게바 바이오파르마, 코포레이션 환자에서 리소좀 산 리파제 결핍증의 치료를 위한 리소좀 산 리파제의 용도
EP2675472A4 (en) 2011-02-15 2014-09-17 Synageva Biopharma Corp METHOD FOR THE TREATMENT OF LACK OF LYSOSOMAL SAURER LIPASE
WO2013023763A1 (en) 2011-08-16 2013-02-21 Instituto Nacional De Tecnologia Agropecuaria (Inta) Bitransgenic bovine
DK2753346T3 (da) 2011-09-07 2020-05-25 Sinai School Medicine Ceramidase og celledifferentiering
WO2013096899A2 (en) 2011-12-23 2013-06-27 Shire Human Genetic Therapies, Inc. Stable formulations for cns delivery of arylsulfatase a
WO2013134530A1 (en) 2012-03-07 2013-09-12 Amicus Therapeutics, Inc. High concentration alpha-glucosidase compositions for the treatment of pompe disease
EP3679923B1 (en) 2012-06-01 2021-08-04 Icahn School of Medicine at Mount Sinai Ceramide levels in the treatment and prevention of infections
EP2956003A2 (en) 2013-02-13 2015-12-23 Laboratoire Français du Fractionnement et des Biotechnologies Proteins with modified glycosylation and methods of production thereof
EP3594231A1 (en) 2013-02-13 2020-01-15 Laboratoire Français du Fractionnement et des Biotechnologies Highly galactosylated anti-tnf-alpha antibodies and uses thereof
CN105189542A (zh) 2013-02-20 2015-12-23 瓦莱里昂治疗有限责任公司 用于治疗庞贝氏症的方法和组合物
JP6832158B2 (ja) 2013-03-14 2021-02-24 アイカーン スクール オブ メディシン アット マウント サイナイ 治療用酸性セラミダーゼ組成物ならびにそれを作製および使用する方法
MA40460A (fr) 2014-08-11 2016-02-18 Shire Human Genetic Therapies Ciblage lysosomial et utilisation correspondante
CN107075468B (zh) 2014-09-30 2021-12-24 阿米库斯治疗学公司 具有增强的碳水化合物的高强度酸性α-葡糖苷酶
MA41022A (fr) 2014-11-24 2017-10-03 Shire Human Genetic Therapies Ciblage lysosomial et utilisations correspondantes
US20170189497A1 (en) 2015-12-30 2017-07-06 Amicus Therapeutics, Inc. Augmented Acid Alpha-Glucosidase For The Treatment Of Pompe Disease
PE20190127A1 (es) 2016-03-30 2019-01-17 Amicus Therapeutics Inc Metodo para seleccionar proteinas recombinantes ricas en m6p
CA3019128A1 (en) 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0279582A3 (en) * 1987-02-17 1989-10-18 Pharming B.V. Dna sequences to target proteins to the mammary gland for efficient secretion
WO1993025567A1 (en) * 1992-06-15 1993-12-23 Gene Pharming Europe B.V. Production of recombinant polypeptides by bovine species and transgenic methods
US5356804A (en) * 1990-10-24 1994-10-18 Mount Sinai School Of Medicine Of The City Of New York Cloning and expression of biologically active human α-galactosidase A
US6118045A (en) * 1995-08-02 2000-09-12 Pharming B.V. Lysosomal proteins produced in the milk of transgenic animals

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DE69628306T2 (de) 2004-04-01
PT845939E (pt) 2003-10-31
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US20070011754A1 (en) 2007-01-11
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WO1997005771A3 (en) 1997-04-03
US6118045A (en) 2000-09-12
US20040003421A1 (en) 2004-01-01
EP0845939A2 (en) 1998-06-10
US20040172665A1 (en) 2004-09-02
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