JP2015523855A5 - - Google Patents
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- JP2015523855A5 JP2015523855A5 JP2015512861A JP2015512861A JP2015523855A5 JP 2015523855 A5 JP2015523855 A5 JP 2015523855A5 JP 2015512861 A JP2015512861 A JP 2015512861A JP 2015512861 A JP2015512861 A JP 2015512861A JP 2015523855 A5 JP2015523855 A5 JP 2015523855A5
- Authority
- JP
- Japan
- Prior art keywords
- oligonucleotide
- nucleotide
- nucleotides
- sequence
- stranded oligonucleotide
- 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
- 108091034117 Oligonucleotide Proteins 0.000 claims description 103
- 125000003729 nucleotide group Chemical group 0.000 claims description 77
- 239000002773 nucleotide Substances 0.000 claims description 68
- 239000005547 deoxyribonucleotide Substances 0.000 claims description 18
- 239000000203 mixture Substances 0.000 claims description 18
- 125000002637 deoxyribonucleotide group Chemical group 0.000 claims description 16
- 101150092476 ABCA1 gene Proteins 0.000 claims description 15
- 101150071783 APOA1 gene Proteins 0.000 claims description 11
- 230000000295 complement effect Effects 0.000 claims description 10
- NYHBQMYGNKIUIF-UUOKFMHZSA-N Guanosine Chemical class C1=NC=2C(=O)NC(N)=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O NYHBQMYGNKIUIF-UUOKFMHZSA-N 0.000 claims description 8
- 101100465401 Saccharomyces cerevisiae (strain ATCC 204508 / S288c) SCL1 gene Proteins 0.000 claims description 8
- 108090000623 proteins and genes Proteins 0.000 claims description 6
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 6
- YBJHBAHKTGYVGT-ZKWXMUAHSA-N (+)-Biotin Chemical group N1C(=O)N[C@@H]2[C@H](CCCCC(=O)O)SC[C@@H]21 YBJHBAHKTGYVGT-ZKWXMUAHSA-N 0.000 claims description 4
- 108700011259 MicroRNAs Proteins 0.000 claims description 4
- 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 claims description 4
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims description 4
- 239000002679 microRNA Substances 0.000 claims description 4
- 201000001320 Atherosclerosis Diseases 0.000 claims description 3
- 208000032928 Dyslipidaemia Diseases 0.000 claims description 3
- 208000017170 Lipid metabolism disease Diseases 0.000 claims description 3
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 2
- 108091028664 Ribonucleotide Proteins 0.000 claims description 2
- 229960002685 biotin Drugs 0.000 claims description 2
- 235000020958 biotin Nutrition 0.000 claims description 2
- 239000011616 biotin Substances 0.000 claims description 2
- 229940104302 cytosine Drugs 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 125000002887 hydroxy group Chemical group [H]O* 0.000 claims description 2
- 239000008194 pharmaceutical composition Substances 0.000 claims description 2
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 2
- 239000002336 ribonucleotide Substances 0.000 claims description 2
- 125000002652 ribonucleotide group Chemical group 0.000 claims description 2
- 238000011144 upstream manufacturing Methods 0.000 claims description 2
- 238000004132 cross linking Methods 0.000 claims 2
- 108091092195 Intron Proteins 0.000 claims 1
- JCAQMQLAHNGVPY-UUOKFMHZSA-N [(2r,3s,4r,5r)-3,4-dihydroxy-5-(2,2,4-trioxo-1h-imidazo[4,5-c][1,2,6]thiadiazin-7-yl)oxolan-2-yl]methyl dihydrogen phosphate Chemical group O[C@@H]1[C@H](O)[C@@H](COP(O)(O)=O)O[C@H]1N1C(NS(=O)(=O)NC2=O)=C2N=C1 JCAQMQLAHNGVPY-UUOKFMHZSA-N 0.000 claims 1
- 230000003247 decreasing effect Effects 0.000 claims 1
- 150000003431 steroids Chemical class 0.000 claims 1
- 238000000034 method Methods 0.000 description 9
- 102100033715 Apolipoprotein A-I Human genes 0.000 description 3
- 102000055510 ATP Binding Cassette Transporter 1 Human genes 0.000 description 2
- 108700005241 ATP Binding Cassette Transporter 1 Proteins 0.000 description 2
- 101000733802 Homo sapiens Apolipoprotein A-I Proteins 0.000 description 2
- 239000007853 buffer solution Substances 0.000 description 1
- 125000002345 steroid group Chemical group 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261647949P | 2012-05-16 | 2012-05-16 | |
| US61/647,949 | 2012-05-16 | ||
| US201361785778P | 2013-03-14 | 2013-03-14 | |
| US61/785,778 | 2013-03-14 | ||
| PCT/US2013/041455 WO2013173647A1 (en) | 2012-05-16 | 2013-05-16 | Compositions and methods for modulating apoa1 and abca1 expression |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2015523855A JP2015523855A (ja) | 2015-08-20 |
| JP2015523855A5 true JP2015523855A5 (enExample) | 2016-06-30 |
Family
ID=49584309
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015512861A Pending JP2015523855A (ja) | 2012-05-16 | 2013-05-16 | Ａｐｏａ１及びａｂｃａ１発現を調節するための組成物及び方法 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20150191722A1 (enExample) |
| EP (1) | EP2849801A4 (enExample) |
| JP (1) | JP2015523855A (enExample) |
| CN (1) | CN104582737A (enExample) |
| AU (1) | AU2013262658A1 (enExample) |
| BR (1) | BR112014028645A2 (enExample) |
| CA (1) | CA2873801A1 (enExample) |
| EA (1) | EA201492119A1 (enExample) |
| WO (1) | WO2013173647A1 (enExample) |
Families Citing this family (34)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3702460A1 (en) | 2010-11-12 | 2020-09-02 | The General Hospital Corporation | Polycomb-associated non-coding rnas |
| US9920317B2 (en) | 2010-11-12 | 2018-03-20 | The General Hospital Corporation | Polycomb-associated non-coding RNAs |
| HK1200484A1 (zh) | 2011-09-14 | 2015-08-07 | Translate Bio Ma, Inc. | 多聚寡核苷酸化合物 |
| CA2866392C (en) | 2012-03-30 | 2023-10-24 | Washington University | Methods for modulating tau expression for reducing seizure and modifying a neurodegenerative syndrome |
| US10837014B2 (en) | 2012-05-16 | 2020-11-17 | Translate Bio Ma, Inc. | Compositions and methods for modulating SMN gene family expression |
| EA201492120A1 (ru) | 2012-05-16 | 2015-10-30 | Рана Терапьютикс, Инк. | Композиции и способы для модулирования экспрессии atp2a2 |
| CN104540946A (zh) | 2012-05-16 | 2015-04-22 | Rana医疗有限公司 | 用于调节utrn表达的组合物和方法 |
| JP2015523854A (ja) | 2012-05-16 | 2015-08-20 | ラナ セラピューティクス インコーポレイテッド | Ｓｍｎ遺伝子ファミリー発現を調節するための組成物及び方法 |
| CN104583402A (zh) | 2012-05-16 | 2015-04-29 | Rana医疗有限公司 | 用于调节mecp2表达的组合物和方法 |
| US10174315B2 (en) | 2012-05-16 | 2019-01-08 | The General Hospital Corporation | Compositions and methods for modulating hemoglobin gene family expression |
| US20150247141A1 (en) | 2012-09-14 | 2015-09-03 | Rana Therapeutics, Inc. | Multimeric oligonucleotide compounds |
| BR112015022156A2 (pt) | 2013-03-14 | 2017-11-14 | Isis Pharmaceuticals Inc | composições e métodos para modular a expressão de tau |
| JP6487913B2 (ja) * | 2013-07-02 | 2019-03-20 | アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． | 成長ホルモン受容体のモジュレータ |
| TWI702046B (zh) | 2013-07-19 | 2020-08-21 | 美商Ionis製藥公司 | 用於調節τ蛋白表現之組合物 |
| JP2016531570A (ja) | 2013-08-16 | 2016-10-13 | ラナ セラピューティクス インコーポレイテッド | ユークロマチン領域を標的とするオリゴヌクレオチド |
| US20170044540A1 (en) * | 2014-04-22 | 2017-02-16 | Mina Therapeutics Limited | Sarna compositions and methods of use |
| WO2015168618A2 (en) | 2014-05-01 | 2015-11-05 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulating growth hormone receptor expression |
| WO2016070060A1 (en) | 2014-10-30 | 2016-05-06 | The General Hospital Corporation | Methods for modulating atrx-dependent gene repression |
| EP3256590A4 (en) * | 2015-02-13 | 2018-10-03 | Translate Bio Ma, Inc. | Targeting oligonucleotides and uses thereof to modulate gene expression |
| WO2016130943A1 (en) | 2015-02-13 | 2016-08-18 | Rana Therapeutics, Inc. | Hybrid oligonucleotides and uses thereof |
| US10900036B2 (en) | 2015-03-17 | 2021-01-26 | The General Hospital Corporation | RNA interactome of polycomb repressive complex 1 (PRC1) |
| MD3519572T2 (ro) | 2016-09-29 | 2024-03-31 | Biogen Ma Inc | Compuși și procedee de reducere a expresiei Tau |
| US11584932B2 (en) | 2016-11-01 | 2023-02-21 | The Research Foundation For The State University Of New York | 5-halouracil-modified microRNAs and their use in the treatment of cancer |
| US20180193270A1 (en) | 2016-11-29 | 2018-07-12 | PureTech Health LLC | Exosomes for delivery of therapeutic agents |
| WO2019048632A1 (en) | 2017-09-08 | 2019-03-14 | Mina Therapeutics Limited | STABILIZED COMPOSITIONS OF SMALL ACTIVATORY RNA (PARNA) OF HNF4A AND METHODS OF USE |
| WO2019048645A1 (en) | 2017-09-08 | 2019-03-14 | Mina Therapeutics Limited | STABILIZED COMPOSITIONS OF SMALL ACTIVATOR RNA (PARNA) FROM CEBPA AND METHODS OF USE |
| LT3700570T (lt) * | 2017-10-23 | 2025-04-10 | Stoke Therapeutics, Inc. | Priešprasmiai oligomerai, skirti būklių ir ligų, pagrįstų nonsens kodono nulemta mrnr degradacija, gydymui |
| BR112020016170A2 (pt) | 2018-02-09 | 2020-12-15 | Genentech, Inc. | Oligonucleotídeos terapêutico e antissenso, conjugado, sal farmaceuticamente aceitável, composição farmacêutica, método in vitro ou in vivo para modular a expressão de tmem106b, método para tratar ou prevenir uma doença, uso do oligonucleotídeo e uso ou método |
| TWI862501B (zh) | 2018-07-03 | 2024-11-21 | 瑞士商赫孚孟拉羅股份公司 | 用於調制 Tau 表現之寡核苷酸 |
| KR102597727B1 (ko) | 2018-09-04 | 2023-11-06 | 프로토타입 앤드 프로덕션 시스템스, 인코포레이티드 | 프린트 모듈 캡핑 스테이션 |
| CN114585633A (zh) | 2019-08-19 | 2022-06-03 | 米纳治疗有限公司 | 寡核苷酸缀合物组合物和使用方法 |
| WO2021262919A2 (en) | 2020-06-26 | 2021-12-30 | The Research Foundation For The State University Of New York | 5-halouracil-modified micrornas and their use in the treatment of cancer |
| US12194057B2 (en) * | 2020-12-17 | 2025-01-14 | Washington University | NXTAR-derived oligonucleotides and uses thereof |
| CN119744197A (zh) | 2022-08-26 | 2025-04-01 | 沃特世科技公司 | 具有无孔有机-无机杂化材料的复合颗粒 |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2000055318A2 (en) * | 1999-03-15 | 2000-09-21 | University Of British Columbia | Abc1 polypeptide and methods and reagents for modulating cholesterol levels |
| CN1474831A (zh) * | 2000-11-20 | 2004-02-11 | ����ŵ˹������ѧ�йܻ� | 膜支架蛋白 |
| AU2002344853A1 (en) * | 2001-06-14 | 2003-01-02 | Active Pass Pharmaceuticals, Inc. | Abca10 transporter |
| US7259150B2 (en) * | 2001-08-07 | 2007-08-21 | Isis Pharmaceuticals, Inc. | Modulation of apolipoprotein (a) expression |
| US20050265927A1 (en) * | 2004-05-17 | 2005-12-01 | Yale University | Intranasal delivery of nucleic acid molecules |
| WO2008103761A2 (en) * | 2007-02-20 | 2008-08-28 | Sequenom, Inc. | Methods and compositions for cancer diagnosis and treatment based on nucleic acid methylation |
| MY188457A (en) * | 2008-10-03 | 2021-12-10 | Opko Curna Llc | Treatment of apolipoprotein-a1 related diseases by inhibition of natural antisense transcript to apolipoprotein-a1 |
| GB0821457D0 (en) * | 2008-11-24 | 2008-12-31 | Trillion Genomics Ltd | Oligonucleotides |
| CN102459596B (zh) * | 2009-05-06 | 2016-09-07 | 库尔纳公司 | 通过针对脂质转运和代谢基因的天然反义转录物的抑制治疗脂质转运和代谢基因相关疾病 |
| AU2011349464B2 (en) * | 2010-12-20 | 2016-07-07 | The General Hospital Corporation | Polycomb-associated non-coding RNAS |
| CN104583398A (zh) * | 2012-05-16 | 2015-04-29 | Rana医疗有限公司 | 用于调节基因表达的组合物和方法 |
-
2013
- 2013-05-16 CN CN201380037631.4A patent/CN104582737A/zh active Pending
- 2013-05-16 AU AU2013262658A patent/AU2013262658A1/en not_active Abandoned
- 2013-05-16 EP EP13790940.4A patent/EP2849801A4/en not_active Withdrawn
- 2013-05-16 EA EA201492119A patent/EA201492119A1/ru unknown
- 2013-05-16 US US14/401,223 patent/US20150191722A1/en not_active Abandoned
- 2013-05-16 BR BR112014028645A patent/BR112014028645A2/pt not_active IP Right Cessation
- 2013-05-16 CA CA2873801A patent/CA2873801A1/en not_active Abandoned
- 2013-05-16 WO PCT/US2013/041455 patent/WO2013173647A1/en not_active Ceased
- 2013-05-16 JP JP2015512861A patent/JP2015523855A/ja active Pending