CA2099503A1 - Alpha-l-iduronidase synthetique et sequences de genes la codant - Google Patents

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

Info

Publication number
CA2099503A1
CA2099503A1 CA002099503A CA2099503A CA2099503A1 CA 2099503 A1 CA2099503 A1 CA 2099503A1 CA 002099503 A CA002099503 A CA 002099503A CA 2099503 A CA2099503 A CA 2099503A CA 2099503 A1 CA2099503 A1 CA 2099503A1
Authority
CA
Canada
Prior art keywords
iduronidase
alpha
sequences encoding
genetic sequences
encoding same
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.)
Granted
Application number
CA002099503A
Other languages
English (en)
Other versions
CA2099503C (fr
Inventor
Hamish Steele Scott
Donald Stewart Anson
Annette Marie Orsborn
Paul Victor Nelson
Peter Roy Clements
Charles Phillip Morris
John Joseph Hopwood
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Womens and Childrens Hospital Adelaide
Original Assignee
Individual
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 Individual filed Critical Individual
Publication of CA2099503A1 publication Critical patent/CA2099503A1/fr
Application granted granted Critical
Publication of CA2099503C publication Critical patent/CA2099503C/fr
Anticipated expiration legal-status Critical
Expired - Lifetime legal-status Critical Current

Links

Classifications

    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/52—Genes encoding for enzymes or proenzymes
    • 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)
    • C12Y302/01076—L-Iduronidase (3.2.1.76)
    • 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
    • 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)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Plant Pathology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
  • Bakery Products And Manufacturing Methods Therefor (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Saccharide Compounds (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
AUPK949091 1991-11-14
AUPK9490/91 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 true CA2099503A1 (fr) 1993-05-15
CA2099503C 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
JP2002514429A (ja) * 1998-05-13 2002-05-21 ハーバー−ユーシーエルエー 組換えα−Ｌ−イズロニダーゼ、その生成及び精製方法及びその欠損により引き起こされる疾病の治療方法
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
WO2012054723A2 (fr) 2010-10-22 2012-04-26 Opko Curna Llc Traitement de maladies associées à l'alpha-l-iduronidase (idua) par inhibition du transcrit antisens endogène de idua
US10940125B2 (en) 2015-09-18 2021-03-09 Duke University Methods and compositions for the treatment of steatosis-associated disorders
EP3576768A4 (fr) * 2017-01-31 2020-11-04 REGENXBIO Inc. Traitement de la mucopolysaccharidose i avec une alpha-l-iduronidase glycosylée entièrement humaine (idua)
WO2019010335A1 (fr) 2017-07-06 2019-01-10 The Trustees Of The University Of Pennsylvania Thérapie génique médiée par aav9 pour traiter la mucopolysaccharidose de type i
EP3684938A1 (fr) 2017-09-22 2020-07-29 The Trustees of the University of Pennsylvania Thérapie génique pour le traitement de la mucopolysaccharidose de type ii

Also Published As

Publication number Publication date
NZ245123A (en) 1995-05-26
AU649897B2 (en) 1994-06-02
WO1993010244A1 (fr) 1993-05-27
JPH06504449A (ja) 1994-05-26
KR930703447A (ko) 1993-11-30
EP0578790A4 (fr) 1994-11-02
CA2099503C (fr) 2007-10-09
AU2914192A (en) 1993-06-15
EP0578790A1 (fr) 1994-01-19

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