CA2099503C - Alpha-l-iduronidase synthetique et sequences de genes la codant - Google Patents

Alpha-l-iduronidase synthetique et sequences de genes la codant Download PDF

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Publication number
CA2099503C
CA2099503C CA002099503A CA2099503A CA2099503C CA 2099503 C CA2099503 C CA 2099503C CA 002099503 A CA002099503 A CA 002099503A CA 2099503 A CA2099503 A CA 2099503A CA 2099503 C CA2099503 C CA 2099503C
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CA
Canada
Prior art keywords
idua
purified
sequence
nucleic acid
amino acid
Prior art date
Legal status (The legal status 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 status listed.)
Expired - Lifetime
Application number
CA002099503A
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English (en)
Other versions
CA2099503A1 (fr
Inventor
Hamish Steele Scott
Donald Stewart Anson
Annette Marie Orsborn
Paul Victor Nelson
Peter Roy Clements
Charles Phillip Morris
John Joseph Hopwood
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Womens and Childrens Hospital Adelaide
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Womens and Childrens Hospital Adelaide
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Application filed by Womens and Childrens Hospital Adelaide filed Critical Womens and Childrens Hospital Adelaide
Publication of CA2099503A1 publication Critical patent/CA2099503A1/fr
Application granted granted Critical
Publication of CA2099503C publication Critical patent/CA2099503C/fr
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Expired - Lifetime legal-status Critical Current

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/52Genes encoding for enzymes or proenzymes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01076L-Iduronidase (3.2.1.76)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Bakery Products And Manufacturing Methods Therefor (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
CA002099503A 1991-11-14 1992-11-12 Alpha-l-iduronidase synthetique et sequences de genes la codant Expired - Lifetime CA2099503C (fr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
AUPK9490/91 1991-11-14
AUPK949091 1991-11-14
PCT/AU1992/000611 WO1993010244A1 (fr) 1991-11-14 1992-11-12 α-L IDURONIDASE SYNTHETIQUE ET SEQUENCES GENETIQUES LA CODANT

Publications (2)

Publication Number Publication Date
CA2099503A1 CA2099503A1 (fr) 1993-05-15
CA2099503C true CA2099503C (fr) 2007-10-09

Family

ID=3775823

Family Applications (1)

Application Number Title Priority Date Filing Date
CA002099503A Expired - Lifetime CA2099503C (fr) 1991-11-14 1992-11-12 Alpha-l-iduronidase synthetique et sequences de genes la codant

Country Status (7)

Country Link
EP (1) EP0578790A4 (fr)
JP (1) JPH06504449A (fr)
KR (1) KR930703447A (fr)
AU (1) AU649897B2 (fr)
CA (1) CA2099503C (fr)
NZ (1) NZ245123A (fr)
WO (1) WO1993010244A1 (fr)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1300323A (zh) * 1998-05-13 2001-06-20 加州大学洛杉矶分校港口研究和教育院 重组α-L-艾杜糖苷酸酶,其生产和纯化的方法以及治疗其缺乏导致的疾病的方法
US6569661B1 (en) 1999-11-12 2003-05-27 Biomarin Pharmaceutical Inc. Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
US6585971B1 (en) 1999-11-12 2003-07-01 Harbor-Ucla Research And Education Institute Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating disease caused by deficiencies thereof
US6426208B1 (en) 1999-11-12 2002-07-30 Harbor-Ucla Research And Education Institute Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
GB0606190D0 (en) 2006-03-28 2006-05-10 Isis Innovation Construct
US8679478B2 (en) 2010-10-04 2014-03-25 Duke University Methods of lysosomal storage disease therapy
CA2815212A1 (fr) 2010-10-22 2012-04-26 Curna, Inc. Traitement de maladies associees a l'alpha-l-iduronidase (idua) par inhibition du transcrit antisens endogene de idua
WO2017049157A1 (fr) 2015-09-18 2017-03-23 Duke University Méthodes et compositions pour le traitement de troubles associés à une stéatose
AU2018216807A1 (en) * 2017-01-31 2019-08-08 Regenxbio Inc. Treatment of mucopolysaccharidosis I with fully-human glycosylated human alpha-l-iduronidase (IDUA)
EP3676385A1 (fr) 2017-07-06 2020-07-08 The Trustees of The University of Pennsylvania Thérapie génique médiée par aav9 pour traiter la mucopolysaccharidose de type i
SG11202002457RA (en) 2017-09-22 2020-04-29 Univ Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii

Also Published As

Publication number Publication date
EP0578790A4 (fr) 1994-11-02
KR930703447A (ko) 1993-11-30
CA2099503A1 (fr) 1993-05-15
EP0578790A1 (fr) 1994-01-19
WO1993010244A1 (fr) 1993-05-27
NZ245123A (en) 1995-05-26
JPH06504449A (ja) 1994-05-26
AU649897B2 (en) 1994-06-02
AU2914192A (en) 1993-06-15

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