CA2099503A1 - Synthetic alpha-l-iduronidase and genetic sequences encoding same - Google Patents

Synthetic alpha-l-iduronidase and genetic sequences encoding same

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
French (fr)
Other versions
CA2099503C (en
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/en
Application granted granted Critical
Publication of CA2099503C publication Critical patent/CA2099503C/en
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)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Plant Pathology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Public Health (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Bakery Products And Manufacturing Methods Therefor (AREA)
  • Saccharide Compounds (AREA)

Abstract

The present invention relates generally to .alpha.-L--iduronidase and to genetic sequences encoding same.
More particularly, the present invention provides an is-olated nucleic acid molecule comprising a sequence of nucleotides which encodes or are complementary to a sequence which encodes a mammalian .alpha.-L-iduronidase or fragment or derivative thereof and to the recombinant enzyme encoded thereby. These molecules are useful in the investigation, diagnosis and treatment of subjects suspected of or suffering from .alpha.-L-iduronidase deficiency.
CA002099503A 1991-11-14 1992-11-12 Synthetic alpha-l-iduronidase and genetic sequences encoding same Expired - Lifetime CA2099503C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
AUPK949091 1991-11-14
AUPK9490/91 1991-11-14
PCT/AU1992/000611 WO1993010244A1 (en) 1991-11-14 1992-11-12 Synthetic alpha-l-iduronidase and genetic sequences encoding same

Publications (2)

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

Family

ID=3775823

Family Applications (1)

Application Number Title Priority Date Filing Date
CA002099503A Expired - Lifetime CA2099503C (en) 1991-11-14 1992-11-12 Synthetic alpha-l-iduronidase and genetic sequences encoding same

Country Status (7)

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

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU4072499A (en) * 1998-05-13 1999-11-29 Harbor-Ucla Recombinant (alpha)-l-iduronidase, methods for producing and purifying the same and methods for treating diseases 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
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
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
US9222088B2 (en) 2010-10-22 2015-12-29 Curna, Inc. Treatment of alpha-L-iduronidase (IDUA) related diseases by inhibition of natural antisense transcript to IDUA
US10940125B2 (en) 2015-09-18 2021-03-09 Duke University Methods and compositions for the treatment of steatosis-associated disorders
SG11201912631PA (en) 2017-07-06 2020-01-30 Univ Pennsylvania Aav9-mediated gene therapy for treating mucopolysaccharidosis type i
BR112020005249A2 (en) 2017-09-22 2020-09-24 The Trustees Of The University Of Pennsylvania gene therapy to treat mucopolysaccharidosis type ii

Also Published As

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

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