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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Women's and Children's Hospital
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Women's and Children's Hospital
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Publication date
Application filed by Women's and Children's Hospital filed Critical Women's and Children's Hospital
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

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    • 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

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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)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Bakery Products And Manufacturing Methods Therefor (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 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
WO1999058691A2 (fr) * 1998-05-13 1999-11-18 Harbor-Ucla (alpha)-l-iduronidase recombinee, procedes de production et de purification de celle-ci et methodes de traitement de maladies dues a des deficits en celle-ci
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
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
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
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
CN103180445B (zh) * 2010-10-22 2018-02-16 库尔纳公司 通过抑制α‑L‑艾杜糖醛酸酶(IDUA)的天然反义转录物而治疗IDUA相关疾病
US10940125B2 (en) 2015-09-18 2021-03-09 Duke University Methods and compositions for the treatment of steatosis-associated disorders
JP2020508289A (ja) * 2017-01-31 2020-03-19 レジェンクスバイオ インコーポレーテッド 完全ヒトグリコシル化ヒトα−Ｌ−イズロニダーゼ（ＩＤＵＡ）を用いたムコ多糖症Ｉ型の治療
CA3068328A1 (fr) 2017-07-06 2019-01-10 The Trustees Of The University Of Pennsylvania Therapie genique mediee 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
KR930703447A (ko) 1993-11-30
AU2914192A (en) 1993-06-15
CA2099503A1 (fr) 1993-05-15
EP0578790A1 (fr) 1994-01-19
JPH06504449A (ja) 1994-05-26
WO1993010244A1 (fr) 1993-05-27
NZ245123A (en) 1995-05-26
AU649897B2 (en) 1994-06-02
EP0578790A4 (fr) 1994-11-02

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