PT2341943T - Composições e métodos para a inibição específica de expressão génica por dsrna que possui modificações - Google Patents
Composições e métodos para a inibição específica de expressão génica por dsrna que possui modificaçõesInfo
- Publication number
- PT2341943T PT2341943T PT09814902T PT09814902T PT2341943T PT 2341943 T PT2341943 T PT 2341943T PT 09814902 T PT09814902 T PT 09814902T PT 09814902 T PT09814902 T PT 09814902T PT 2341943 T PT2341943 T PT 2341943T
- Authority
- PT
- Portugal
- Prior art keywords
- compositions
- methods
- gene expression
- specific inhibition
- dsrna
- Prior art date
Links
- 102000040650 (ribonucleotides)n+m Human genes 0.000 title 1
- 108091032973 (ribonucleotides)n+m Proteins 0.000 title 1
- 230000014509 gene expression Effects 0.000 title 1
- 230000005764 inhibitory process Effects 0.000 title 1
- 238000000034 method Methods 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
- 238000012986 modification Methods 0.000 title 1
- 230000004048 modification Effects 0.000 title 1
Classifications
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- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
- A61P35/02—Antineoplastic agents specific for leukemia
-
- 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
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
-
- 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
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- 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
- C12N2310/14—Type of nucleic acid interfering N.A.
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/331—Universal or degenerate base
-
- 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
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; 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
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/50—Methods for regulating/modulating their activity
- C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/50—Methods for regulating/modulating their activity
- C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Virology (AREA)
- Oncology (AREA)
- Hematology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US13673608P | 2008-09-22 | 2008-09-22 | |
| US13674108P | 2008-09-22 | 2008-09-22 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PT2341943T true PT2341943T (pt) | 2019-02-06 |
Family
ID=42040058
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PT09814902T PT2341943T (pt) | 2008-09-22 | 2009-09-17 | Composições e métodos para a inibição específica de expressão génica por dsrna que possui modificações |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US20100331389A1 (pt) |
| EP (1) | EP2341943B1 (pt) |
| JP (3) | JP2012504389A (pt) |
| AU (1) | AU2009293636A1 (pt) |
| CA (1) | CA2738625C (pt) |
| DK (1) | DK2341943T3 (pt) |
| ES (1) | ES2708944T3 (pt) |
| PT (1) | PT2341943T (pt) |
| WO (1) | WO2010033225A2 (pt) |
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| CN103987847B (zh) | 2011-10-18 | 2017-06-16 | 迪克纳制药公司 | 胺阳离子脂质及其用途 |
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| HUE038146T2 (hu) * | 2013-05-22 | 2018-09-28 | Alnylam Pharmaceuticals Inc | Serpina1 IRNS készítmények és eljárások alkalmazásukra |
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| US10150965B2 (en) | 2013-12-06 | 2018-12-11 | Dicerna Pharmaceuticals, Inc. | Methods and compositions for the specific inhibition of transthyretin (TTR) by double-stranded RNA |
| DK3204497T3 (da) | 2014-10-10 | 2020-05-25 | Dicerna Pharmaceuticals Inc | Terapeutisk hæmning af lactatdehydrogenase og midler dertil |
| EP3234132B1 (en) * | 2014-12-15 | 2019-07-10 | Dicerna Pharmaceuticals, Inc. | Ligand-modified double-stranded nucleic acids |
| US10036017B2 (en) | 2015-02-17 | 2018-07-31 | Dicerna Pharmaceuticals, Inc. | Methods and compositions for the specific inhibition of complement component 5(C5) by double-stranded RNA |
| KR102350647B1 (ko) | 2016-09-02 | 2022-01-14 | 다이서나 파마수이티컬, 인크. | 4'-포스페이트 유사체 및 이를 포함하는 올리고뉴클레오타이드 |
| TW202313978A (zh) | 2016-11-23 | 2023-04-01 | 美商阿尼拉製藥公司 | 絲胺酸蛋白酶抑制因子Ａ１ ｉＲＮＡ組成物及其使用方法 |
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| US10994025B2 (en) | 2017-05-12 | 2021-05-04 | Massachusetts Institute Of Technology | Argonaute protein-double stranded RNA complexes and uses related thereto |
| IL295086A (en) | 2017-06-29 | 2022-09-01 | Dicerna Pharmaceuticals Inc | Compositions and methods for inhibiting expression of hmgb1 |
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| KR20200144100A (ko) | 2018-04-13 | 2020-12-28 | 다이서나 파마수이티컬, 인크. | Tm-증가 뉴클레오타이드로 변형된 이중-가닥 핵산 억제제 분자 |
| CN113454221A (zh) * | 2018-12-12 | 2021-09-28 | 迪克纳制药公司 | 含有三元环的双链核酸抑制剂分子 |
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| EP1943265B1 (en) * | 2005-10-01 | 2012-09-12 | Charles Stout | Regulatable fusion promoters |
| US8329888B2 (en) | 2006-03-23 | 2012-12-11 | Santaris Pharma A/S | Small internally segmented interfering RNA |
| JP5876637B2 (ja) * | 2006-10-18 | 2016-03-02 | マリーナ バイオテック，インコーポレイテッド | ニックまたはギャップの入った核酸分子およびそれらの使用 |
-
2009
- 2009-09-17 PT PT09814902T patent/PT2341943T/pt unknown
- 2009-09-17 WO PCT/US2009/005214 patent/WO2010033225A2/en active Application Filing
- 2009-09-17 JP JP2011527823A patent/JP2012504389A/ja active Pending
- 2009-09-17 DK DK09814902.4T patent/DK2341943T3/en active
- 2009-09-17 CA CA2738625A patent/CA2738625C/en active Active
- 2009-09-17 US US12/586,281 patent/US20100331389A1/en not_active Abandoned
- 2009-09-17 EP EP09814902.4A patent/EP2341943B1/en active Active
- 2009-09-17 ES ES09814902T patent/ES2708944T3/es active Active
- 2009-09-17 AU AU2009293636A patent/AU2009293636A1/en not_active Abandoned
-
2012
- 2012-01-09 US US13/346,331 patent/US20130041010A1/en not_active Abandoned
-
2015
- 2015-07-02 JP JP2015133329A patent/JP6295232B2/ja active Active
-
2016
- 2016-12-13 JP JP2016240842A patent/JP2017079761A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| JP2012504389A (ja) | 2012-02-23 |
| US20100331389A1 (en) | 2010-12-30 |
| WO2010033225A2 (en) | 2010-03-25 |
| AU2009293636A1 (en) | 2010-03-25 |
| CA2738625C (en) | 2017-12-12 |
| EP2341943A2 (en) | 2011-07-13 |
| WO2010033225A3 (en) | 2012-01-05 |
| JP2015221042A (ja) | 2015-12-10 |
| JP2017079761A (ja) | 2017-05-18 |
| DK2341943T3 (en) | 2019-02-25 |
| JP6295232B2 (ja) | 2018-03-14 |
| EP2341943A4 (en) | 2013-05-22 |
| US20130041010A1 (en) | 2013-02-14 |
| EP2341943B1 (en) | 2018-11-07 |
| ES2708944T3 (es) | 2019-04-12 |
| CA2738625A1 (en) | 2010-03-25 |
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