BR9808606A - Materiais e métodos para tratamento de doenças com ribozimas - Google Patents
Materiais e métodos para tratamento de doenças com ribozimasInfo
- Publication number
- BR9808606A BR9808606A BR9808606-5A BR9808606A BR9808606A BR 9808606 A BR9808606 A BR 9808606A BR 9808606 A BR9808606 A BR 9808606A BR 9808606 A BR9808606 A BR 9808606A
- Authority
- BR
- Brazil
- Prior art keywords
- methods
- materials
- ribozymes
- treating diseases
- mrna
- Prior art date
Links
- 238000000034 method Methods 0.000 title abstract 4
- 102000053642 Catalytic RNA Human genes 0.000 title abstract 2
- 108090000994 Catalytic RNA Proteins 0.000 title abstract 2
- 201000010099 disease Diseases 0.000 title abstract 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title abstract 2
- 108091092562 ribozyme Proteins 0.000 title abstract 2
- 108020004999 messenger RNA Proteins 0.000 abstract 2
- 208000017442 Retinal disease Diseases 0.000 abstract 1
- 208000007014 Retinitis pigmentosa Diseases 0.000 abstract 1
- 230000008030 elimination Effects 0.000 abstract 1
- 238000003379 elimination reaction Methods 0.000 abstract 1
- 238000002560 therapeutic procedure 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
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1136—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against growth factors, growth regulators, cytokines, lymphokines or hormones
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
-
- 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
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
-
- 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
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1138—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
- C12N2310/111—Antisense spanning the whole gene, or a large part of it
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
- C12N2310/121—Hammerhead
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
- C12N2310/122—Hairpin
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
- C12N2310/127—DNAzymes
-
- 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
- C12N2799/00—Uses of viruses
- C12N2799/02—Uses of viruses as vector
- C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
- C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Chemical & Material Sciences (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Endocrinology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicinal Chemistry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Virology (AREA)
- Cardiology (AREA)
- Vascular Medicine (AREA)
- Urology & Nephrology (AREA)
- Heart & Thoracic Surgery (AREA)
- Ophthalmology & Optometry (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Enzymes And Modification Thereof (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US4449297P | 1997-04-21 | 1997-04-21 | |
| US4614797P | 1997-05-09 | 1997-05-09 | |
| PCT/US1998/007968 WO1998048009A2 (en) | 1997-04-21 | 1998-04-21 | Materials and methods for ribozyme treatment of retinal diseases |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR9808606A true BR9808606A (pt) | 2000-05-23 |
Family
ID=26721633
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR9808606-5A BR9808606A (pt) | 1997-04-21 | 1998-04-21 | Materiais e métodos para tratamento de doenças com ribozimas |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US6225291B1 (https=) |
| EP (1) | EP0977841A2 (https=) |
| JP (1) | JP2001523959A (https=) |
| AU (1) | AU735788B2 (https=) |
| BR (1) | BR9808606A (https=) |
| CA (1) | CA2287508A1 (https=) |
| NZ (1) | NZ500427A (https=) |
| WO (1) | WO1998048009A2 (https=) |
Families Citing this family (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6225291B1 (en) | 1997-04-21 | 2001-05-01 | University Of Florida | Rod opsin mRNA-specific ribozyme compositions and methods for the treatment of retinal diseases |
| DE19956568A1 (de) * | 1999-01-30 | 2000-08-17 | Roland Kreutzer | Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens |
| US6485976B1 (en) | 1999-04-30 | 2002-11-26 | City Of Hope | Use of adeno-associated virus (AAV) to deliver genes |
| JP2002542805A (ja) * | 1999-04-30 | 2002-12-17 | ユニバーシティ オブ フロリダ | アデノ随伴ウイルス送達リボザイム組成物および使用方法 |
| US7829693B2 (en) * | 1999-11-24 | 2010-11-09 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of a target gene |
| US8546143B2 (en) | 2001-01-09 | 2013-10-01 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of a target gene |
| US20030003582A1 (en) * | 2001-05-08 | 2003-01-02 | Tranzyme, Inc. | Trans-viral vector mediated gene transfer to the retina |
| CA2482904A1 (en) | 2002-04-18 | 2003-10-23 | Lynkeus Biotech Gmbh | Means and methods for the specific modulation of target genes in the cns and the eye and methods for their identification |
| US7148342B2 (en) | 2002-07-24 | 2006-12-12 | The Trustees Of The University Of Pennyslvania | Compositions and methods for sirna inhibition of angiogenesis |
| ES2334125T3 (es) | 2002-11-04 | 2010-03-05 | University Of Massachusetts | Interferencia de arn especifico de alelos. |
| US8680063B2 (en) | 2003-09-12 | 2014-03-25 | University Of Massachusetts | RNA interference for the treatment of gain-of-function disorders |
| EP2239328A3 (en) | 2005-08-18 | 2011-01-05 | Alnylam Pharmaceuticals Inc. | Methods and compositions for treating neurological disease |
| US8257969B2 (en) * | 2007-04-12 | 2012-09-04 | The Provost Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth | Genetic suppression and replacement |
| JP5832293B2 (ja) | 2008-12-04 | 2015-12-16 | オプコ ファーマシューティカルズ、エルエルシー | 血管新生促進ｖｅｇｆイソ型を選択的に抑制する組成物および方法 |
| US8450473B2 (en) | 2009-04-30 | 2013-05-28 | The Research Foundation Of State University Of New York | Compositions and methods for therapy of macular degeneration |
| HRP20190144T1 (hr) | 2010-04-23 | 2019-03-22 | University Of Florida Research Foundation, Inc. | Pripravci raav-gvanilat ciklaze i postupci za liječenje leberove urođene amauroze-1 (lca1) |
| IL248102B (en) | 2014-05-02 | 2022-07-01 | Genzyme Corp | aav vectors for gene therapy of the central nervous system and retina |
| EP4012035B1 (en) | 2014-09-16 | 2024-11-06 | Genzyme Corporation | Adeno-associated viral vectors for treating myocilin (myoc) glaucoma |
| PT4012035T (pt) | 2014-09-16 | 2025-02-11 | Genzyme Corp | Vetores virais adenoassociados para tratar o glaucoma de miocilina (mioc) |
| BR112017017178A2 (pt) | 2015-02-26 | 2018-04-03 | Ionis Pharmaceuticals Inc | moduladores específicos de alelo de rodopsina de p23h |
| US11653636B2 (en) * | 2019-11-15 | 2023-05-23 | University Of Ulsan Foundation For Industry Cooperation | Method of making a rat model of retinal degeneration and rat model made thereby |
| US20210261625A1 (en) | 2020-01-29 | 2021-08-26 | Genzyme Corporation | Modified adeno-associated viral capsid proteins for ocular gene therapy and methods of use thereof |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4987071A (en) | 1986-12-03 | 1991-01-22 | University Patents, Inc. | RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods |
| US5037746A (en) | 1986-12-03 | 1991-08-06 | University Patents, Inc. | RNA ribozyme polymerases, and methods |
| US5116742A (en) | 1986-12-03 | 1992-05-26 | University Patents, Inc. | RNA ribozyme restriction endoribonucleases and methods |
| US5646020A (en) | 1992-05-14 | 1997-07-08 | Ribozyme Pharmaceuticals, Inc. | Hammerhead ribozymes for preferred targets |
| US5498539A (en) * | 1992-07-02 | 1996-03-12 | Daiichi Pharmaceutical Co., Ltd. | Bovine endothelial nitric oxide synthase nucleic acids |
| US5639655A (en) | 1993-01-19 | 1997-06-17 | Ribozyme Pharmaceuticals, Inc. | PML-RARA targeted ribozymes |
| US6410322B1 (en) * | 1993-07-27 | 2002-06-25 | Hybridon Inc | Antisense oligonucleotide inhibition of vascular endothelial growth factor expression |
| US5646031A (en) | 1995-05-16 | 1997-07-08 | Northern Illinois University | SArMV and sCYMVI hairpin ribozymes |
| GB9519299D0 (en) | 1995-09-21 | 1995-11-22 | Farrar Gwyneth J | Genetic strategy |
| GB9604449D0 (en) | 1996-03-01 | 1996-05-01 | Farrar Gwyneth J | Genetic strategy ii |
| CA2287495A1 (en) * | 1997-04-21 | 1998-10-29 | University Of Florida | Materials and methods for treatment of retinal diseases |
| US6225291B1 (en) | 1997-04-21 | 2001-05-01 | University Of Florida | Rod opsin mRNA-specific ribozyme compositions and methods for the treatment of retinal diseases |
-
1998
- 1998-04-21 US US09/063,667 patent/US6225291B1/en not_active Expired - Lifetime
- 1998-04-21 CA CA002287508A patent/CA2287508A1/en not_active Abandoned
- 1998-04-21 EP EP98918493A patent/EP0977841A2/en not_active Withdrawn
- 1998-04-21 NZ NZ500427A patent/NZ500427A/xx unknown
- 1998-04-21 AU AU71406/98A patent/AU735788B2/en not_active Ceased
- 1998-04-21 WO PCT/US1998/007968 patent/WO1998048009A2/en not_active Ceased
- 1998-04-21 JP JP54625498A patent/JP2001523959A/ja not_active Ceased
- 1998-04-21 BR BR9808606-5A patent/BR9808606A/pt not_active IP Right Cessation
Also Published As
| Publication number | Publication date |
|---|---|
| WO1998048009A2 (en) | 1998-10-29 |
| EP0977841A2 (en) | 2000-02-09 |
| AU735788B2 (en) | 2001-07-12 |
| CA2287508A1 (en) | 1998-10-29 |
| US6225291B1 (en) | 2001-05-01 |
| AU7140698A (en) | 1998-11-13 |
| WO1998048009A3 (en) | 1999-03-25 |
| JP2001523959A (ja) | 2001-11-27 |
| NZ500427A (en) | 2002-12-20 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| B08F | Application dismissed because of non-payment of annual fees [chapter 8.6 patent gazette] | ||
| B08K | Patent lapsed as no evidence of payment of the annual fee has been furnished to inpi [chapter 8.11 patent gazette] |
Free format text: REFERENTE AO DESPACHO PUBLICADO NA RPI 1937 DE 19/02/2008 |