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)
- 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)
- Veterinary Medicine (AREA)
- Vascular Medicine (AREA)
- Urology & Nephrology (AREA)
- Ophthalmology & Optometry (AREA)
- Cardiology (AREA)
- Heart & Thoracic Surgery (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 (OSRAM) |
| EP (1) | EP0977841A2 (OSRAM) |
| JP (1) | JP2001523959A (OSRAM) |
| AU (1) | AU735788B2 (OSRAM) |
| BR (1) | BR9808606A (OSRAM) |
| CA (1) | CA2287508A1 (OSRAM) |
| NZ (1) | NZ500427A (OSRAM) |
| WO (1) | WO1998048009A2 (OSRAM) |
Families Citing this family (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| BR9808606A (pt) | 1997-04-21 | 2000-05-23 | Univ Florida | Materiais e métodos para tratamento de doenças com ribozimas |
| 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 |
| WO2000066780A2 (en) * | 1999-04-30 | 2000-11-09 | University Of Florida | Adeno-associated virus-delivered ribozyme compositions and methods of use |
| 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 |
| JP5578388B2 (ja) | 2002-04-18 | 2014-08-27 | オプコ ファーマシューティカルズ、エルエルシー | 中枢神経システム及び／若しくは眼の細胞及び組織中の遺伝子の特異的阻害のための手段と方法 |
| US7148342B2 (en) | 2002-07-24 | 2006-12-12 | The Trustees Of The University Of Pennyslvania | Compositions and methods for sirna inhibition of angiogenesis |
| ATE443764T1 (de) | 2002-11-04 | 2009-10-15 | Univ Massachusetts | Allelspezifische rna-interferenz |
| US8680063B2 (en) | 2003-09-12 | 2014-03-25 | University Of Massachusetts | RNA interference for the treatment of gain-of-function disorders |
| JP2009504782A (ja) | 2005-08-18 | 2009-02-05 | アルナイラム ファーマシューティカルズ インコーポレイテッド | 神経疾患を治療するための方法および組成物 |
| CA2683469C (en) | 2007-04-12 | 2019-11-26 | The Provost, Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth | Genetic suppression and replacement |
| US8470792B2 (en) | 2008-12-04 | 2013-06-25 | Opko Pharmaceuticals, Llc. | Compositions and methods for selective inhibition of VEGF |
| 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 |
| KR20200047642A (ko) | 2010-04-23 | 2020-05-07 | 유니버시티 오브 플로리다 리서치 파운데이션, 인크. | 레베르 선천성 흑내장-1(lca1)의 치료를 위한 재조합 아데노-관련된 바이러스-구아닐레이트 사이클라제 조성물 및 방법 |
| EP3137497B2 (en) | 2014-05-02 | 2025-03-05 | Genzyme Corporation | Aav vectors for retinal and cns gene therapy |
| WO2016044478A1 (en) | 2014-09-16 | 2016-03-24 | Genzyme Corporation | Adeno-associated viral vectors for treating myocilin (myoc) glaucoma |
| EP4012035B1 (en) | 2014-09-16 | 2024-11-06 | Genzyme Corporation | Adeno-associated viral vectors for treating myocilin (myoc) glaucoma |
| US10426789B2 (en) | 2015-02-26 | 2019-10-01 | Ionis Pharmaceuticals, Inc. | Allele specific modulators of P23H rhodopsin |
| 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 |
| JP2023513004A (ja) | 2020-01-29 | 2023-03-30 | ジェンザイム・コーポレーション | 眼科遺伝子療法のための改変されたアデノ随伴ウイルスカプシドタンパク質およびその使用方法 |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5037746A (en) | 1986-12-03 | 1991-08-06 | University Patents, Inc. | RNA ribozyme polymerases, and methods |
| US4987071A (en) | 1986-12-03 | 1991-01-22 | University Patents, Inc. | RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases 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 |
| BR9809284A (pt) | 1997-04-21 | 2004-08-03 | Univ Florida | Construto e processo para expressão de polinucleotìdeo em altos nìveis |
| BR9808606A (pt) | 1997-04-21 | 2000-05-23 | Univ Florida | Materiais e métodos para tratamento de doenças com ribozimas |
-
1998
- 1998-04-21 BR BR9808606-5A patent/BR9808606A/pt not_active IP Right Cessation
- 1998-04-21 EP EP98918493A patent/EP0977841A2/en not_active Withdrawn
- 1998-04-21 CA CA002287508A patent/CA2287508A1/en not_active Abandoned
- 1998-04-21 US US09/063,667 patent/US6225291B1/en not_active Expired - Lifetime
- 1998-04-21 JP JP54625498A patent/JP2001523959A/ja not_active Ceased
- 1998-04-21 NZ NZ500427A patent/NZ500427A/xx unknown
- 1998-04-21 WO PCT/US1998/007968 patent/WO1998048009A2/en not_active Ceased
- 1998-04-21 AU AU71406/98A patent/AU735788B2/en not_active Ceased
Also Published As
| Publication number | Publication date |
|---|---|
| EP0977841A2 (en) | 2000-02-09 |
| AU7140698A (en) | 1998-11-13 |
| WO1998048009A2 (en) | 1998-10-29 |
| CA2287508A1 (en) | 1998-10-29 |
| JP2001523959A (ja) | 2001-11-27 |
| AU735788B2 (en) | 2001-07-12 |
| NZ500427A (en) | 2002-12-20 |
| WO1998048009A3 (en) | 1999-03-25 |
| US6225291B1 (en) | 2001-05-01 |
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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 |