JP2006517793A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2006517793A5 JP2006517793A5 JP2006501691A JP2006501691A JP2006517793A5 JP 2006517793 A5 JP2006517793 A5 JP 2006517793A5 JP 2006501691 A JP2006501691 A JP 2006501691A JP 2006501691 A JP2006501691 A JP 2006501691A JP 2006517793 A5 JP2006517793 A5 JP 2006517793A5
- Authority
- JP
- Japan
- Prior art keywords
- gene
- target gene
- pei
- cell
- single stranded
- 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.)
- Pending
Links
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 8
- 102000040650 (ribonucleotides)n+m Human genes 0.000 claims description 5
- 210000004027 cell Anatomy 0.000 claims description 5
- 108020004999 messenger RNA Proteins 0.000 claims description 3
- 210000004962 mammalian cell Anatomy 0.000 claims description 2
- 238000000034 method Methods 0.000 claims 18
- 108090000623 proteins and genes Proteins 0.000 claims 14
- 108091027075 5S-rRNA precursor Proteins 0.000 claims 5
- 238000012228 RNA interference-mediated gene silencing Methods 0.000 claims 3
- 108091028664 Ribonucleotide Proteins 0.000 claims 3
- 238000007385 chemical modification Methods 0.000 claims 3
- 230000000295 complement effect Effects 0.000 claims 3
- 230000009368 gene silencing by RNA Effects 0.000 claims 3
- 239000002336 ribonucleotide Substances 0.000 claims 3
- 125000002652 ribonucleotide group Chemical group 0.000 claims 3
- 108090000695 Cytokines Proteins 0.000 claims 1
- 108091000054 Prion Proteins 0.000 claims 1
- 230000000711 cancerogenic effect Effects 0.000 claims 1
- 231100000315 carcinogenic Toxicity 0.000 claims 1
- 210000003527 eukaryotic cell Anatomy 0.000 claims 1
- 230000004048 modification Effects 0.000 claims 1
- 238000012986 modification Methods 0.000 claims 1
- 125000003729 nucleotide group Chemical group 0.000 claims 1
- 230000001575 pathological effect Effects 0.000 claims 1
- 230000001105 regulatory effect Effects 0.000 claims 1
- 150000004713 phosphodiesters Chemical class 0.000 description 5
- 101710163270 Nuclease Proteins 0.000 description 3
- 108091034117 Oligonucleotide Proteins 0.000 description 3
- 230000000692 anti-sense effect Effects 0.000 description 3
- 230000005764 inhibitory process Effects 0.000 description 2
- 108020004707 nucleic acids Proteins 0.000 description 2
- 102000039446 nucleic acids Human genes 0.000 description 2
- 150000007523 nucleic acids Chemical class 0.000 description 2
- 238000001890 transfection Methods 0.000 description 2
- 241000699800 Cricetinae Species 0.000 description 1
- 230000008836 DNA modification Effects 0.000 description 1
- 102000000033 Purinergic Receptors Human genes 0.000 description 1
- 108010080192 Purinergic Receptors Proteins 0.000 description 1
- 108020004459 Small interfering RNA Proteins 0.000 description 1
- RYYWUUFWQRZTIU-UHFFFAOYSA-N Thiophosphoric acid Chemical class OP(O)(S)=O RYYWUUFWQRZTIU-UHFFFAOYSA-N 0.000 description 1
- JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Polymers Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 description 1
- 238000013459 approach Methods 0.000 description 1
- 230000015572 biosynthetic process Effects 0.000 description 1
- 230000015556 catabolic process Effects 0.000 description 1
- 230000004700 cellular uptake Effects 0.000 description 1
- 239000003795 chemical substances by application Substances 0.000 description 1
- 239000002299 complementary DNA Substances 0.000 description 1
- 238000006731 degradation reaction Methods 0.000 description 1
- 238000011161 development Methods 0.000 description 1
- 230000029087 digestion Effects 0.000 description 1
- 230000000694 effects Effects 0.000 description 1
- 238000002474 experimental method Methods 0.000 description 1
- 238000010348 incorporation Methods 0.000 description 1
- 239000003112 inhibitor Substances 0.000 description 1
- 150000002632 lipids Chemical class 0.000 description 1
- 238000004519 manufacturing process Methods 0.000 description 1
- -1 methoxyethyl Chemical group 0.000 description 1
- 239000002777 nucleoside Substances 0.000 description 1
- 150000003833 nucleoside derivatives Chemical class 0.000 description 1
- 210000001672 ovary Anatomy 0.000 description 1
- 230000001681 protective effect Effects 0.000 description 1
- 230000002269 spontaneous effect Effects 0.000 description 1
- 238000003786 synthesis reaction Methods 0.000 description 1
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 description 1
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US44397703P | 2003-01-31 | 2003-01-31 | |
| PCT/EP2004/000897 WO2004066989A1 (en) | 2003-01-31 | 2004-01-30 | Down-regulation of target-gene with pei/single-stranded oligoribonucleotide comp lexes |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2006517793A JP2006517793A (ja) | 2006-08-03 |
| JP2006517793A5 true JP2006517793A5 (https=) | 2007-02-15 |
Family
ID=32825395
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2006501691A Pending JP2006517793A (ja) | 2003-01-31 | 2004-01-30 | スクリーニングアッセイ |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20060135453A1 (https=) |
| EP (1) | EP1592412A1 (https=) |
| JP (1) | JP2006517793A (https=) |
| CN (1) | CN1744886A (https=) |
| BR (1) | BRPI0407107A (https=) |
| CA (1) | CA2514536A1 (https=) |
| WO (1) | WO2004066989A1 (https=) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20060030534A1 (en) * | 2002-09-04 | 2006-02-09 | Gabriele Dorn | Treatment of neurological disorders by dsrna administration |
| EP2287301A3 (en) * | 2004-08-31 | 2011-11-02 | Sylentis S.A.U. | Methods and compositions to inhibit P2X7 receptor expression |
| JP5802389B2 (ja) * | 2007-12-13 | 2015-10-28 | ポリプラス−トランスフェクション | 合成ポリマーを用いた遺伝子サイレンシングに活性な核酸を送達する手段 |
| EA037448B1 (ru) * | 2012-06-08 | 2021-03-30 | Этрис Гмбх | Способ доставки в легкие терапевтической мрнк с pei и фармацевтическая композиция |
| WO2018010815A1 (en) | 2016-07-15 | 2018-01-18 | Biontech Rna Pharmaceuticals Gmbh | Formulation for administration of rna |
| CN111601605A (zh) * | 2018-01-11 | 2020-08-28 | 生物技术Rna制药有限公司 | 用于施用rna的制剂 |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO1996013599A1 (en) * | 1994-11-01 | 1996-05-09 | Winfried Wels | Nucleic acid transfer system |
| US6353055B1 (en) * | 1994-11-18 | 2002-03-05 | Supratek Pharma Inc. | Polynucleotide compositions |
| US20030143204A1 (en) * | 2001-07-27 | 2003-07-31 | Lewis David L. | Inhibition of RNA function by delivery of inhibitors to animal cells |
| FR2793414B1 (fr) * | 1999-05-10 | 2003-05-23 | Centre Nat Rech Scient | Conjugue acide nucleique-anticorps pour delivrer un acide nucleique etranger dans les cellules |
| DE10015906B4 (de) * | 2000-03-30 | 2006-09-07 | Ibfb Pharma Gmbh | Gerichteter Gentransfer in Thy-1-positive Zellen |
| US6586524B2 (en) * | 2001-07-19 | 2003-07-01 | Expression Genetics, Inc. | Cellular targeting poly(ethylene glycol)-grafted polymeric gene carrier |
| US20030198627A1 (en) * | 2001-09-01 | 2003-10-23 | Gert-Jan Arts | siRNA knockout assay method and constructs |
| JP2005508396A (ja) * | 2001-11-02 | 2005-03-31 | イントラディグム、コーポレイション | 核酸送達ビヒクルのための治療方法 |
| AU2003207708A1 (en) * | 2002-02-20 | 2003-09-09 | Sirna Therapeutics, Inc. | Rna interference mediated inhibition of map kinase genes |
| AU2003217009A1 (en) * | 2002-03-07 | 2003-09-16 | Biocompatibles Uk Limited | Compositions of polymers |
| WO2004014933A1 (en) * | 2002-08-07 | 2004-02-19 | University Of Massachusetts | Compositions for rna interference and methods of use thereof |
| EP1575574A4 (en) * | 2002-10-30 | 2007-11-07 | Blood Res Center | METHOD FOR TREATING AND PREVENTING APOPTOSIS-BASED DISEASES BY MEANS OF RNA INTERFERENCE AGENTS |
-
2004
- 2004-01-30 BR BR0407107-7A patent/BRPI0407107A/pt not_active IP Right Cessation
- 2004-01-30 EP EP04706627A patent/EP1592412A1/en not_active Ceased
- 2004-01-30 WO PCT/EP2004/000897 patent/WO2004066989A1/en not_active Ceased
- 2004-01-30 CN CNA2004800033048A patent/CN1744886A/zh active Pending
- 2004-01-30 US US10/543,362 patent/US20060135453A1/en not_active Abandoned
- 2004-01-30 CA CA002514536A patent/CA2514536A1/en not_active Abandoned
- 2004-01-30 JP JP2006501691A patent/JP2006517793A/ja active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| EP2341943B1 (en) | Compositions and methods for the specific inhibition of gene expression by dsrna possessing modifications | |
| AU2011282243B2 (en) | Modulation of nuclear-retained RNA | |
| EP2444494B1 (en) | Rna antagonist compounds for the modulation of pcsk9 | |
| EP3119888B1 (en) | Compositions for modulating ataxin 2 expression | |
| CA2640058C (en) | Oligomeric compounds and compositions for the use in modulation of micrornas | |
| EP3444350B1 (en) | Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded rna | |
| US20180127754A1 (en) | Bivalent antisense oligonucleotides | |
| Pasternak et al. | Unlocked nucleic acid–an RNA modification with broad potential | |
| JP2024056820A (ja) | Ｓｃｎ９ａ発現を調節するためのオリゴヌクレオチド | |
| EP3995580A2 (en) | Compounds and methods for modulation of dystrophia myotonica-protein kinase (dmpk) expression | |
| CA2994089A1 (en) | Antisense gapmer oligonucleotides | |
| EP3757214A1 (en) | Compositions for modulating sod-1 expression | |
| JP2012531888A (ja) | 非対称二本鎖ｒｎａによる特異的阻害のための、方法および組成物 | |
| JP2015502365A5 (https=) | ||
| EP4636085A2 (en) | Compositions for modulating mecp2 expression | |
| WO2016019063A1 (en) | Compositions and methods for modulating tau expression | |
| US20110288147A1 (en) | Compositions and methods for the specific inhibition of gene expression by DSRNA containing a tetraloop | |
| CA3088522A1 (en) | Modulators of dnm2 expression | |
| HK1214301A1 (zh) | 治療epas1相關疾病的有機組合物 | |
| JP2006517793A5 (https=) | ||
| WO2008011473A2 (en) | Compositions and their uses directed to hbxip | |
| TW202400786A (zh) | 具有RNAi活性的化學修飾寡核苷酸 | |
| TW202309286A (zh) | 作為新穎基因靜默技術的非對稱短雙股ｄｎａ及其應用 | |
| CA3221923A1 (en) | Short duplex dna as a novel gene silencing technology and use thereof | |
| Muñoz-Alarcón et al. | Modulating Anti‐MicroRNA‐21 Activity and Specificity Using Oligonucleotide Derivatives and Length Optimization |