JP2008514242A5 - - Google Patents
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- Publication number
- JP2008514242A5 JP2008514242A5 JP2007534866A JP2007534866A JP2008514242A5 JP 2008514242 A5 JP2008514242 A5 JP 2008514242A5 JP 2007534866 A JP2007534866 A JP 2007534866A JP 2007534866 A JP2007534866 A JP 2007534866A JP 2008514242 A5 JP2008514242 A5 JP 2008514242A5
- Authority
- JP
- Japan
- Prior art keywords
- sirna
- sirna molecule
- modification
- molecule
- modified
- 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
- 108020004459 Small interfering RNA Proteins 0.000 claims description 53
- 125000002652 ribonucleotide group Chemical group 0.000 claims description 7
- 108091028664 Ribonucleotide Proteins 0.000 claims description 6
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims description 6
- 239000002336 ribonucleotide Substances 0.000 claims description 6
- 125000003729 nucleotide group Chemical group 0.000 claims description 4
- 235000012000 cholesterol Nutrition 0.000 claims description 3
- 239000002773 nucleotide Substances 0.000 claims description 3
- 150000003230 pyrimidines Chemical class 0.000 claims description 3
- 230000010076 replication Effects 0.000 claims description 2
- 230000004048 modification Effects 0.000 claims 13
- 238000012986 modification Methods 0.000 claims 13
- 239000003446 ligand Substances 0.000 claims 5
- 102000005962 receptors Human genes 0.000 claims 5
- 108020003175 receptors Proteins 0.000 claims 5
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical compound O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims 3
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical group NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims 3
- CZPWVGJYEJSRLH-UHFFFAOYSA-N Pyrimidine Chemical compound C1=CN=CN=C1 CZPWVGJYEJSRLH-UHFFFAOYSA-N 0.000 claims 2
- 125000001153 fluoro group Chemical group F* 0.000 claims 2
- 102000007330 LDL Lipoproteins Human genes 0.000 claims 1
- 108010007622 LDL Lipoproteins Proteins 0.000 claims 1
- 102000006382 Ribonucleases Human genes 0.000 claims 1
- 108010083644 Ribonucleases Proteins 0.000 claims 1
- 239000000427 antigen Substances 0.000 claims 1
- 102000036639 antigens Human genes 0.000 claims 1
- 108091007433 antigens Proteins 0.000 claims 1
- 230000015556 catabolic process Effects 0.000 claims 1
- 229940104302 cytosine Drugs 0.000 claims 1
- 238000006731 degradation reaction Methods 0.000 claims 1
- 230000002401 inhibitory effect Effects 0.000 claims 1
- -1 methoxyethyl Chemical group 0.000 claims 1
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims 1
- 125000001436 propyl group Chemical group [H]C([*])([H])C([H])([H])C([H])([H])[H] 0.000 claims 1
- 229940035893 uracil Drugs 0.000 claims 1
- 230000029812 viral genome replication Effects 0.000 claims 1
- 210000004027 cell Anatomy 0.000 description 11
- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 10
- 108060001084 Luciferase Proteins 0.000 description 9
- 230000000694 effects Effects 0.000 description 9
- 239000005089 Luciferase Substances 0.000 description 8
- 108091027544 Subgenomic mRNA Proteins 0.000 description 5
- 230000001965 increasing effect Effects 0.000 description 5
- 102100031181 Glyceraldehyde-3-phosphate dehydrogenase Human genes 0.000 description 4
- 108091027305 Heteroduplex Proteins 0.000 description 4
- 231100000673 dose–response relationship Toxicity 0.000 description 4
- 108020004445 glyceraldehyde-3-phosphate dehydrogenase Proteins 0.000 description 4
- 230000003247 decreasing effect Effects 0.000 description 3
- 230000001939 inductive effect Effects 0.000 description 3
- 238000003670 luciferase enzyme activity assay Methods 0.000 description 3
- 210000002966 serum Anatomy 0.000 description 3
- WAVYAFBQOXCGSZ-UHFFFAOYSA-N 2-fluoropyrimidine Chemical group FC1=NC=CC=N1 WAVYAFBQOXCGSZ-UHFFFAOYSA-N 0.000 description 2
- 108091006112 ATPases Proteins 0.000 description 2
- 102000057290 Adenosine Triphosphatases Human genes 0.000 description 2
- 101000600434 Homo sapiens Putative uncharacterized protein encoded by MIR7-3HG Proteins 0.000 description 2
- 241000699670 Mus sp. Species 0.000 description 2
- 102100037401 Putative uncharacterized protein encoded by MIR7-3HG Human genes 0.000 description 2
- 108091030071 RNAI Proteins 0.000 description 2
- 241000315672 SARS coronavirus Species 0.000 description 2
- 230000009368 gene silencing by RNA Effects 0.000 description 2
- 210000003494 hepatocyte Anatomy 0.000 description 2
- 210000005229 liver cell Anatomy 0.000 description 2
- 239000003550 marker Substances 0.000 description 2
- 239000000203 mixture Substances 0.000 description 2
- 238000006467 substitution reaction Methods 0.000 description 2
- 238000001890 transfection Methods 0.000 description 2
- 102000040650 (ribonucleotides)n+m Human genes 0.000 description 1
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 description 1
- 108020003589 5' Untranslated Regions Proteins 0.000 description 1
- 108020004414 DNA Proteins 0.000 description 1
- 241000255581 Drosophila <fruit fly, genus> Species 0.000 description 1
- 108090000790 Enzymes Proteins 0.000 description 1
- 102000004190 Enzymes Human genes 0.000 description 1
- 208000034454 F12-related hereditary angioedema with normal C1Inh Diseases 0.000 description 1
- 108090000331 Firefly luciferases Proteins 0.000 description 1
- 101000907904 Homo sapiens Endoribonuclease Dicer Proteins 0.000 description 1
- 108700026244 Open Reading Frames Proteins 0.000 description 1
- 238000004458 analytical method Methods 0.000 description 1
- 238000003556 assay Methods 0.000 description 1
- 238000003149 assay kit Methods 0.000 description 1
- 239000005547 deoxyribonucleotide Substances 0.000 description 1
- 125000002637 deoxyribonucleotide group Chemical group 0.000 description 1
- 238000010586 diagram Methods 0.000 description 1
- 238000011156 evaluation Methods 0.000 description 1
- 238000003682 fluorination reaction Methods 0.000 description 1
- 230000034659 glycolysis Effects 0.000 description 1
- 206010073071 hepatocellular carcinoma Diseases 0.000 description 1
- 231100000844 hepatocellular carcinoma Toxicity 0.000 description 1
- 208000016861 hereditary angioedema type 3 Diseases 0.000 description 1
- 102000051308 human DICER1 Human genes 0.000 description 1
- 238000001727 in vivo Methods 0.000 description 1
- 238000011534 incubation Methods 0.000 description 1
- 230000005764 inhibitory process Effects 0.000 description 1
- 238000002347 injection Methods 0.000 description 1
- 239000007924 injection Substances 0.000 description 1
- 239000002502 liposome Substances 0.000 description 1
- 108020004999 messenger RNA Proteins 0.000 description 1
- 230000008685 targeting Effects 0.000 description 1
- 231100000331 toxic Toxicity 0.000 description 1
- 230000002588 toxic effect Effects 0.000 description 1
- 230000035899 viability Effects 0.000 description 1
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US61495504P | 2004-10-01 | 2004-10-01 | |
| PCT/US2005/035493 WO2006039656A2 (en) | 2004-10-01 | 2005-09-30 | Modified small interfering rna molecules and methods of use |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012054494A Division JP2012105686A (ja) | 2004-10-01 | 2012-03-12 | 改変型の小さい干渉ｒｎａ分子および使用方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2008514242A JP2008514242A (ja) | 2008-05-08 |
| JP2008514242A5 true JP2008514242A5 (https=) | 2008-10-02 |
Family
ID=36143139
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2007534866A Pending JP2008514242A (ja) | 2004-10-01 | 2005-09-30 | 改変型の小さい干渉ｒｎａ分子および使用方法 |
| JP2012054494A Pending JP2012105686A (ja) | 2004-10-01 | 2012-03-12 | 改変型の小さい干渉ｒｎａ分子および使用方法 |
| JP2012193264A Pending JP2012255024A (ja) | 2004-10-01 | 2012-09-03 | 改変型の小さい干渉ｒｎａ分子および使用方法 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012054494A Pending JP2012105686A (ja) | 2004-10-01 | 2012-03-12 | 改変型の小さい干渉ｒｎａ分子および使用方法 |
| JP2012193264A Pending JP2012255024A (ja) | 2004-10-01 | 2012-09-03 | 改変型の小さい干渉ｒｎａ分子および使用方法 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US8138161B2 (https=) |
| EP (1) | EP1796732B1 (https=) |
| JP (3) | JP2008514242A (https=) |
| CA (1) | CA2581651C (https=) |
| ES (1) | ES2441791T3 (https=) |
| WO (1) | WO2006039656A2 (https=) |
Families Citing this family (23)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2715289C (en) | 2008-02-11 | 2019-12-24 | Rxi Pharmaceuticals Corporation | Modified rnai polynucleotides and uses thereof |
| US8815818B2 (en) | 2008-07-18 | 2014-08-26 | Rxi Pharmaceuticals Corporation | Phagocytic cell delivery of RNAI |
| EP2342340A1 (en) * | 2008-09-22 | 2011-07-13 | Rxi Pharmaceuticals Corporation | Rna interference in skin indications |
| WO2010059226A2 (en) | 2008-11-19 | 2010-05-27 | Rxi Pharmaceuticals Corporation | Inhibition of map4k4 through rnai |
| WO2010078536A1 (en) | 2009-01-05 | 2010-07-08 | Rxi Pharmaceuticals Corporation | Inhibition of pcsk9 through rnai |
| US9745574B2 (en) | 2009-02-04 | 2017-08-29 | Rxi Pharmaceuticals Corporation | RNA duplexes with single stranded phosphorothioate nucleotide regions for additional functionality |
| RU2615143C2 (ru) | 2010-03-24 | 2017-04-04 | Адвирна | Самодоставляющие PHKi соединения уменьшенного размера |
| CN103200945B (zh) | 2010-03-24 | 2016-07-06 | 雷克西制药公司 | 眼部症候中的rna干扰 |
| EP3560503B1 (en) | 2010-03-24 | 2021-11-17 | Phio Pharmaceuticals Corp. | Rna interference in dermal and fibrotic indications |
| US9029341B2 (en) * | 2010-08-17 | 2015-05-12 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of hepatitis B virus (HBV) gene expression using short interfering nucleic acid (siNA) |
| CN106061488B (zh) | 2013-12-02 | 2021-04-09 | 菲奥医药公司 | 癌症的免疫治疗 |
| WO2015168108A2 (en) | 2014-04-28 | 2015-11-05 | Rxi Pharmaceuticals Corporation | Methods for treating cancer using nucleic targeting mdm2 or mycn |
| US10900039B2 (en) | 2014-09-05 | 2021-01-26 | Phio Pharmaceuticals Corp. | Methods for treating aging and skin disorders using nucleic acids targeting Tyr or MMP1 |
| GB201418135D0 (en) * | 2014-10-14 | 2014-11-26 | London School Hygiene & Tropical Medicine | Anti-viral agent |
| EP3862005A1 (en) | 2015-07-06 | 2021-08-11 | Phio Pharmaceuticals Corp. | Nucleic acid molecules targeting superoxide dismutase 1 (sod1) |
| US10808247B2 (en) | 2015-07-06 | 2020-10-20 | Phio Pharmaceuticals Corp. | Methods for treating neurological disorders using a synergistic small molecule and nucleic acids therapeutic approach |
| US20170137821A1 (en) | 2015-07-17 | 2017-05-18 | Arcturus Therapeutics, Inc. | Molecules and agents for treating hepatitis b virus |
| CA2996722A1 (en) | 2015-07-17 | 2017-01-26 | Arcturus Therapeutics, Inc. | Compositions and agents against hepatitis b virus and uses thereof |
| WO2017070151A1 (en) | 2015-10-19 | 2017-04-27 | Rxi Pharmaceuticals Corporation | Reduced size self-delivering nucleic acid compounds targeting long non-coding rna |
| CA3043768A1 (en) | 2016-11-29 | 2018-06-07 | PureTech Health LLC | Exosomes for delivery of therapeutic agents |
| US12544344B2 (en) | 2017-04-19 | 2026-02-10 | Phio Pharmaceuticals Corp. | Topical delivery of nucleic acid compounds |
| WO2019014359A2 (en) * | 2017-07-12 | 2019-01-17 | The Scripps Research Institute | POLYMERASE CHAIN TRANSCRIPTION (PCT): EXPONENTIAL SYNTHESIS OF RNA AND MODIFIED RNA |
| US11425125B2 (en) * | 2020-06-24 | 2022-08-23 | Google Llc | Shared resource identification |
Family Cites Families (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20030206887A1 (en) * | 1992-05-14 | 2003-11-06 | David Morrissey | RNA interference mediated inhibition of hepatitis B virus (HBV) using short interfering nucleic acid (siNA) |
| US5610054A (en) * | 1992-05-14 | 1997-03-11 | Ribozyme Pharmaceuticals, Inc. | Enzymatic RNA molecule targeted against Hepatitis C virus |
| US6174868B1 (en) * | 1992-09-10 | 2001-01-16 | Isis Pharmaceuticals, Inc. | Compositions and methods for treatment of hepatitis C virus-associated diseases |
| EP0662128B1 (en) * | 1992-09-28 | 2005-11-23 | Chiron Corporation | Methods and compositions for controlling translation of hcv proteins |
| US6107027A (en) * | 1994-12-14 | 2000-08-22 | University Of Washington | Ribozymes for treating hepatitis C |
| US6133246A (en) * | 1997-08-13 | 2000-10-17 | Isis Pharmaceuticals Inc. | Antisense oligonucleotide compositions and methods for the modulation of JNK proteins |
| US6506559B1 (en) * | 1997-12-23 | 2003-01-14 | Carnegie Institute Of Washington | Genetic inhibition by double-stranded RNA |
| JP2002536405A (ja) * | 1999-02-11 | 2002-10-29 | イーデンランド、インコーポレイテッド | ウイルス感染症の治療方法 |
| US8202979B2 (en) * | 2002-02-20 | 2012-06-19 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid |
| EP1386004A4 (en) * | 2001-04-05 | 2005-02-16 | Ribozyme Pharm Inc | MODULATION OF GENE EXPRESSION ASSOCIATED WITH INFLAMMATORY PROLIFERATION AND GROWTH OF NEURITIES BY METHODS INVOLVING NUCLEIC ACID |
| US20040209831A1 (en) * | 2002-02-20 | 2004-10-21 | Mcswiggen James | RNA interference mediated inhibition of hepatitis C virus (HCV) gene expression using short interfering nucleic acid (siNA) |
| US20050209180A1 (en) * | 2001-05-18 | 2005-09-22 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of hepatitis C virus (HCV) expression using short interfering nucleic acid (siNA) |
| DK2280070T3 (en) * | 2001-07-23 | 2015-08-24 | Univ Leland Stanford Junior | Methods and compositions for use in RNAi-mediated inhibition of gene expression in mammals |
| US20030219823A1 (en) * | 2001-09-07 | 2003-11-27 | Alsobrook John P. | Therapeutic polypeptides, nucleic acids encoding same, and methods of use |
| US20080207542A1 (en) | 2002-03-26 | 2008-08-28 | Sirna Therapeutics, Inc. | RNA inteference mediated inhibition of hepatitis C virus (HVC) gene expression using short interfering nucleic acid (siNA) |
| US20030191075A1 (en) * | 2002-02-22 | 2003-10-09 | Cook Phillip Dan | Method of using modified oligonucleotides for hepatic delivery |
| AU2003224725A1 (en) | 2002-03-20 | 2003-10-08 | Brigham And Women's Hospital, Inc. | Hiv therapeutic |
| US7148342B2 (en) | 2002-07-24 | 2006-12-12 | The Trustees Of The University Of Pennyslvania | Compositions and methods for sirna inhibition of angiogenesis |
| AU2003261231A1 (en) * | 2002-07-26 | 2004-02-16 | Chiron Corporation | Modified small interfering rna molecules and methods of use |
| NZ540779A (en) | 2002-11-01 | 2008-05-30 | Univ Pennsylvania | Compositions and methods for siRNA inhibition of HIF-1 alpha |
| AU2003295600A1 (en) * | 2002-11-14 | 2004-06-15 | Dharmacon, Inc. | Functional and hyperfunctional sirna |
| JP2007524349A (ja) | 2003-01-16 | 2007-08-30 | ザ・トラスティーズ・オブ・ザ・ユニバーシティ・オブ・ペンシルバニア | ＩＣＡＭ−１のｓｉＲＮＡ阻害のための組成物及び方法 |
| DE10302421A1 (de) * | 2003-01-21 | 2004-07-29 | Ribopharma Ag | Doppelsträngige Ribonukleinsäure mit verbesserter Wirksamkeit |
| US20050085528A1 (en) * | 2003-10-17 | 2005-04-21 | Pfizer Inc. | Parmaceutical |
| JP2007536937A (ja) * | 2004-05-12 | 2007-12-20 | カメル・カリーリ | 霊長類ポリオーマウイルス遺伝子のｓｉＲＮＡ干渉用組成物および方法 |
| AR066984A1 (es) * | 2007-06-15 | 2009-09-23 | Novartis Ag | Inhibicion de la expresion de la subunidad alfa del canal epitelial de sodio (enac) por medio de arni (arn de interferencia) |
| EP3000885B1 (en) * | 2009-12-18 | 2018-07-25 | Arrowhead Pharmaceuticals, Inc. | Organic compositions to treat hsf1-related diseases |
-
2005
- 2005-09-30 WO PCT/US2005/035493 patent/WO2006039656A2/en not_active Ceased
- 2005-09-30 US US11/664,008 patent/US8138161B2/en not_active Expired - Fee Related
- 2005-09-30 ES ES05808592.9T patent/ES2441791T3/es not_active Expired - Lifetime
- 2005-09-30 EP EP05808592.9A patent/EP1796732B1/en not_active Expired - Lifetime
- 2005-09-30 CA CA2581651A patent/CA2581651C/en not_active Expired - Fee Related
- 2005-09-30 JP JP2007534866A patent/JP2008514242A/ja active Pending
-
2012
- 2012-03-12 JP JP2012054494A patent/JP2012105686A/ja active Pending
- 2012-09-03 JP JP2012193264A patent/JP2012255024A/ja active Pending
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