BRPI0922355A8 - Ácidos nucléicos, métodos para reduzir a expressão de um gene em uma célula in vitro, uso do ácido nucléico, complexo de rna e uso do complexo de rna - Google Patents
Ácidos nucléicos, métodos para reduzir a expressão de um gene em uma célula in vitro, uso do ácido nucléico, complexo de rna e uso do complexo de rnaInfo
- Publication number
- BRPI0922355A8 BRPI0922355A8 BRPI0922355A BRPI0922355A BRPI0922355A8 BR PI0922355 A8 BRPI0922355 A8 BR PI0922355A8 BR PI0922355 A BRPI0922355 A BR PI0922355A BR PI0922355 A BRPI0922355 A BR PI0922355A BR PI0922355 A8 BRPI0922355 A8 BR PI0922355A8
- Authority
- BR
- Brazil
- Prior art keywords
- rna complex
- rna
- gene
- vitro
- cell
- Prior art date
Links
- 108020004707 nucleic acids Proteins 0.000 title abstract 4
- 102000039446 nucleic acids Human genes 0.000 title abstract 4
- 150000007523 nucleic acids Chemical class 0.000 title abstract 4
- 230000014509 gene expression Effects 0.000 title abstract 3
- 238000000338 in vitro Methods 0.000 title abstract 2
- 238000000034 method Methods 0.000 title abstract 2
- 108090000623 proteins and genes Proteins 0.000 title abstract 2
- 108091032973 (ribonucleotides)n+m Proteins 0.000 abstract 6
- 108091081021 Sense strand Proteins 0.000 abstract 2
- 108020004459 Small interfering RNA Proteins 0.000 abstract 2
- 239000004055 small Interfering RNA Substances 0.000 abstract 2
- 102000040650 (ribonucleotides)n+m Human genes 0.000 abstract 1
- 230000000692 anti-sense effect Effects 0.000 abstract 1
- 125000004029 hydroxymethyl group Chemical group [H]OC([H])([H])* 0.000 abstract 1
- 230000001225 therapeutic effect Effects 0.000 abstract 1
- 230000001052 transient effect Effects 0.000 abstract 1
Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
- A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
- A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/06—Antihyperlipidemics
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
- A61P31/14—Antivirals for RNA viruses
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
- A61P31/14—Antivirals for RNA viruses
- A61P31/16—Antivirals for RNA viruses for influenza or rhinoviruses
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
- A61P31/20—Antivirals for DNA viruses
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
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- 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
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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- C—CHEMISTRY; METALLURGY
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- 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
- C12N15/1131—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 viruses
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- 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
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
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- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
- G01N2800/00—Detection or diagnosis of diseases
- G01N2800/10—Musculoskeletal or connective tissue disorders
- G01N2800/101—Diffuse connective tissue disease, e.g. Sjögren, Wegener's granulomatosis
- G01N2800/102—Arthritis; Rheumatoid arthritis, i.e. inflammation of peripheral joints
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Virology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
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- Communicable Diseases (AREA)
- Oncology (AREA)
- Physical Education & Sports Medicine (AREA)
- Rheumatology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Obesity (AREA)
- Gastroenterology & Hepatology (AREA)
- Epidemiology (AREA)
- Immunology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Pain & Pain Management (AREA)
Abstract
ÁCIDOS NUCLÉICOS, MÉTODOS PARA REDUZIR A EXPRESSÃO DE UM GENE EM UMA CÉLULA IN VITRO, USO DO ÁCIDO NUCLÉICO, COMPLEXO DE RNA e USO DO COMPLEXO DE RNA Esta divulgação fornece complexos de RNA com fita dupla tendo um ou mais nucleomonômeros substituído(s) com hidroximetilna fita passageira (ou fita senso) de um complexo de RNA. Complexos de RNA do presente pedido podem ser úteis para aplicações terapêuticas, aplicações de diagnóstico ou de aplicações de pesquisa. Complexos de RNA incluem complexos de RNA de interferência curtos (siRNA) capazes de modular a expressão de gene compreendendo uma fita antisenso e uma fita passageira contínua ou descontínua ("fita senso"). Além disso, um ou mais nucleomonômero substituído(s) com hidroximetil da presente divulgação podem ser posicionados na terminação 3', na terminação-5', em ambos as terminações 3' e 5'.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US11962408P | 2008-12-03 | 2008-12-03 | |
| US25208509P | 2009-10-15 | 2009-10-15 | |
| PCT/US2009/066610 WO2010065756A2 (en) | 2008-12-03 | 2009-12-03 | Usirna complexes |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| BRPI0922355A2 BRPI0922355A2 (pt) | 2017-10-24 |
| BRPI0922355A8 true BRPI0922355A8 (pt) | 2017-12-12 |
Family
ID=42111396
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BRPI0922355A BRPI0922355A8 (pt) | 2008-12-03 | 2009-12-03 | Ácidos nucléicos, métodos para reduzir a expressão de um gene em uma célula in vitro, uso do ácido nucléico, complexo de rna e uso do complexo de rna |
Country Status (12)
| Country | Link |
|---|---|
| US (2) | US9340789B2 (pt) |
| EP (1) | EP2370577B1 (pt) |
| JP (3) | JP5816556B2 (pt) |
| KR (1) | KR20110097915A (pt) |
| CN (2) | CN102239259A (pt) |
| AU (2) | AU2009322290B2 (pt) |
| BR (1) | BRPI0922355A8 (pt) |
| CA (1) | CA2744093A1 (pt) |
| IL (1) | IL212941A (pt) |
| SG (2) | SG10201500318SA (pt) |
| WO (1) | WO2010065756A2 (pt) |
| ZA (1) | ZA201104540B (pt) |
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| US20150025122A1 (en) | 2009-10-12 | 2015-01-22 | Larry J. Smith | Methods and Compositions for Modulating Gene Expression Using Oligonucleotide Based Drugs Administered in vivo or in vitro |
| IL314045A (en) | 2013-12-12 | 2024-09-01 | Alnylam Pharmaceuticals Inc | Complement component irna compositions and methods of use thereof |
| US10119136B2 (en) | 2014-01-09 | 2018-11-06 | Alnylam Pharmaceuticals, Inc. | RNAi agents modified at the 4′-C position |
| KR102592370B1 (ko) | 2014-02-11 | 2023-10-25 | 알닐람 파마슈티칼스 인코포레이티드 | 케토헥소키나제(KHK) iRNA 조성물 및 그의 사용 방법 |
| US9856475B2 (en) | 2014-03-25 | 2018-01-02 | Arcturus Therapeutics, Inc. | Formulations for treating amyloidosis |
| CA2946719C (en) * | 2014-03-25 | 2023-09-26 | Arcturus Therapeutics, Inc. | Una oligomers having reduced off-target effects in gene silencing |
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- 2009-12-03 CA CA2744093A patent/CA2744093A1/en not_active Abandoned
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| CA2744093A1 (en) | 2010-06-10 |
| AU2009322290B2 (en) | 2016-06-16 |
| SG10201500318SA (en) | 2015-03-30 |
| JP2015078196A (ja) | 2015-04-23 |
| CN104673798B (zh) | 2018-03-20 |
| WO2010065756A2 (en) | 2010-06-10 |
| JP5816556B2 (ja) | 2015-11-18 |
| WO2010065756A3 (en) | 2010-11-25 |
| AU2016228247A1 (en) | 2016-10-06 |
| KR20110097915A (ko) | 2011-08-31 |
| EP2370577B1 (en) | 2019-08-28 |
| CN102239259A (zh) | 2011-11-09 |
| IL212941A0 (en) | 2011-07-31 |
| US20160215293A1 (en) | 2016-07-28 |
| US20110313020A1 (en) | 2011-12-22 |
| JP2012510297A (ja) | 2012-05-10 |
| EP2370577A2 (en) | 2011-10-05 |
| US9340789B2 (en) | 2016-05-17 |
| IL212941A (en) | 2016-05-31 |
| CN104673798A (zh) | 2015-06-03 |
| SG171879A1 (en) | 2011-07-28 |
| BRPI0922355A2 (pt) | 2017-10-24 |
| AU2009322290A1 (en) | 2010-06-10 |
| ZA201104540B (en) | 2012-08-29 |
| JP2017140030A (ja) | 2017-08-17 |
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