CA2099503A1 - Alpha-l-iduronidase synthetique et sequences de genes la codant - Google Patents
Alpha-l-iduronidase synthetique et sequences de genes la codantInfo
- 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
Links
- 230000002068 genetic effect Effects 0.000 title abstract 2
- 102000004627 Iduronidase Human genes 0.000 title 1
- 108010003381 Iduronidase Proteins 0.000 title 1
- 102000004190 Enzymes Human genes 0.000 abstract 1
- 108090000790 Enzymes Proteins 0.000 abstract 1
- 230000000295 complement effect Effects 0.000 abstract 1
- 230000007812 deficiency Effects 0.000 abstract 1
- 238000003745 diagnosis Methods 0.000 abstract 1
- 239000012634 fragment Substances 0.000 abstract 1
- 238000011835 investigation Methods 0.000 abstract 1
- 150000007523 nucleic acids Chemical class 0.000 abstract 1
- 102000039446 nucleic acids Human genes 0.000 abstract 1
- 108020004707 nucleic acids Proteins 0.000 abstract 1
- 239000002773 nucleotide Substances 0.000 abstract 1
- 125000003729 nucleotide group Chemical group 0.000 abstract 1
- 238000011282 treatment Methods 0.000 abstract 1
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)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Plant Pathology (AREA)
- Veterinary Medicine (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Pharmacology & Pharmacy (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)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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)
| 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 |
| 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 |
| 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 |
| KR101865433B1 (ko) | 2010-10-22 | 2018-07-13 | 큐알엔에이, 인크. | 알파-l 이두로니다아제 (idua)에 대한 자연 안티센스 전사체의 저해에 의한 idua 관련된 질환의 치료 |
| WO2017049157A1 (fr) | 2015-09-18 | 2017-03-23 | Duke University | Méthodes et compositions pour le traitement de troubles associés à une stéatose |
| KR20250022877A (ko) * | 2017-01-31 | 2025-02-17 | 리젠엑스바이오 인크. | 완전히-인간 글리코실화된 인간 알파-l-이두로니다아제(idua)를 이용한 뮤코다당증 1형의 치료 |
| 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 |
| KR102883778B1 (ko) | 2017-09-22 | 2025-11-12 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Ii형 점액다당류증의 치료를 위한 유전자 요법 |
-
1992
- 1992-11-12 CA CA002099503A patent/CA2099503C/fr not_active Expired - Lifetime
- 1992-11-12 AU AU29141/92A patent/AU649897B2/en not_active Ceased
- 1992-11-12 JP JP5508825A patent/JPH06504449A/ja active Pending
- 1992-11-12 WO PCT/AU1992/000611 patent/WO1993010244A1/fr not_active Ceased
- 1992-11-12 EP EP9292923074A patent/EP0578790A4/fr not_active Withdrawn
- 1992-11-12 KR KR1019930702107A patent/KR930703447A/ko not_active Withdrawn
- 1992-11-13 NZ NZ245123A patent/NZ245123A/en not_active IP Right Cessation
Also Published As
| Publication number | Publication date |
|---|---|
| KR930703447A (ko) | 1993-11-30 |
| AU2914192A (en) | 1993-06-15 |
| JPH06504449A (ja) | 1994-05-26 |
| EP0578790A1 (fr) | 1994-01-19 |
| WO1993010244A1 (fr) | 1993-05-27 |
| CA2099503C (fr) | 2007-10-09 |
| AU649897B2 (en) | 1994-06-02 |
| EP0578790A4 (fr) | 1994-11-02 |
| NZ245123A (en) | 1995-05-26 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| EEER | Examination request | ||
| MKEX | Expiry |