JP2013518604A5 - - Google Patents
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- JP2013518604A5 JP2013518604A5 JP2012552932A JP2012552932A JP2013518604A5 JP 2013518604 A5 JP2013518604 A5 JP 2013518604A5 JP 2012552932 A JP2012552932 A JP 2012552932A JP 2012552932 A JP2012552932 A JP 2012552932A JP 2013518604 A5 JP2013518604 A5 JP 2013518604A5
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- JP
- Japan
- Prior art keywords
- compound
- modified
- disease
- wing
- nucleoside
- 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.)
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- 150000001875 compounds Chemical class 0.000 claims 24
- 108091034117 Oligonucleotide Proteins 0.000 claims 16
- 239000002777 nucleoside Substances 0.000 claims 16
- 125000003835 nucleoside group Chemical group 0.000 claims 7
- 150000003833 nucleoside derivatives Chemical class 0.000 claims 6
- 239000002773 nucleotide Substances 0.000 claims 6
- 125000003729 nucleotide group Chemical group 0.000 claims 6
- 239000000074 antisense oligonucleotide Substances 0.000 claims 5
- 238000012230 antisense oligonucleotides Methods 0.000 claims 5
- -1 bicyclic nucleoside Chemical class 0.000 claims 3
- 235000021092 sugar substitutes Nutrition 0.000 claims 3
- 239000003765 sweetening agent Substances 0.000 claims 3
- 108700028369 Alleles Proteins 0.000 claims 2
- 125000002619 bicyclic group Chemical group 0.000 claims 2
- 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 2
- 201000010099 disease Diseases 0.000 claims 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 2
- 208000011403 Alexander disease Diseases 0.000 claims 1
- 208000024827 Alzheimer disease Diseases 0.000 claims 1
- 208000019901 Anxiety disease Diseases 0.000 claims 1
- 102000014461 Ataxins Human genes 0.000 claims 1
- 108010078286 Ataxins Proteins 0.000 claims 1
- 206010003658 Atrial Fibrillation Diseases 0.000 claims 1
- 206010006187 Breast cancer Diseases 0.000 claims 1
- 208000026310 Breast neoplasm Diseases 0.000 claims 1
- 206010068597 Bulbospinal muscular atrophy congenital Diseases 0.000 claims 1
- 208000031229 Cardiomyopathies Diseases 0.000 claims 1
- 208000024172 Cardiovascular disease Diseases 0.000 claims 1
- 206010007764 Cataract subcapsular Diseases 0.000 claims 1
- 206010008025 Cerebellar ataxia Diseases 0.000 claims 1
- 208000006545 Chronic Obstructive Pulmonary Disease Diseases 0.000 claims 1
- 208000004117 Congenital Myasthenic Syndromes Diseases 0.000 claims 1
- 206010010356 Congenital anomaly Diseases 0.000 claims 1
- 208000020406 Creutzfeldt Jacob disease Diseases 0.000 claims 1
- 208000003407 Creutzfeldt-Jakob Syndrome Diseases 0.000 claims 1
- 208000010859 Creutzfeldt-Jakob disease Diseases 0.000 claims 1
- 208000005819 Dystonia Musculorum Deformans Diseases 0.000 claims 1
- 208000003807 Graves Disease Diseases 0.000 claims 1
- 208000015023 Graves' disease Diseases 0.000 claims 1
- 208000023105 Huntington disease Diseases 0.000 claims 1
- 206010020590 Hypercalciuria Diseases 0.000 claims 1
- 208000035150 Hypercholesterolemia Diseases 0.000 claims 1
- 206010020772 Hypertension Diseases 0.000 claims 1
- 206010023230 Joint stiffness Diseases 0.000 claims 1
- 208000027747 Kennedy disease Diseases 0.000 claims 1
- 208000002569 Machado-Joseph Disease Diseases 0.000 claims 1
- 208000001145 Metabolic Syndrome Diseases 0.000 claims 1
- 208000021384 Obsessive-Compulsive disease Diseases 0.000 claims 1
- 208000018737 Parkinson disease Diseases 0.000 claims 1
- 208000005764 Peripheral Arterial Disease Diseases 0.000 claims 1
- 208000030831 Peripheral arterial occlusive disease Diseases 0.000 claims 1
- 102000029797 Prion Human genes 0.000 claims 1
- 108091000054 Prion Proteins 0.000 claims 1
- 206010060862 Prostate cancer Diseases 0.000 claims 1
- 208000000236 Prostatic Neoplasms Diseases 0.000 claims 1
- 208000007014 Retinitis pigmentosa Diseases 0.000 claims 1
- 208000009415 Spinocerebellar Ataxias Diseases 0.000 claims 1
- 208000036834 Spinocerebellar ataxia type 3 Diseases 0.000 claims 1
- 206010067584 Type 1 diabetes mellitus Diseases 0.000 claims 1
- 208000006269 X-Linked Bulbo-Spinal Atrophy Diseases 0.000 claims 1
- 201000000690 abdominal obesity-metabolic syndrome Diseases 0.000 claims 1
- 201000006288 alpha thalassemia Diseases 0.000 claims 1
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 claims 1
- 230000036506 anxiety Effects 0.000 claims 1
- 208000006673 asthma Diseases 0.000 claims 1
- 201000004562 autosomal dominant cerebellar ataxia Diseases 0.000 claims 1
- 210000003591 cerebellar nuclei Anatomy 0.000 claims 1
- 201000001883 cholelithiasis Diseases 0.000 claims 1
- 235000012000 cholesterol Nutrition 0.000 claims 1
- 230000000295 complement effect Effects 0.000 claims 1
- 230000007547 defect Effects 0.000 claims 1
- 201000006061 fatal familial insomnia Diseases 0.000 claims 1
- 206010073071 hepatocellular carcinoma Diseases 0.000 claims 1
- 231100000844 hepatocellular carcinoma Toxicity 0.000 claims 1
- 208000019423 liver disease Diseases 0.000 claims 1
- 230000036244 malformation Effects 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 230000004048 modification Effects 0.000 claims 1
- 238000012986 modification Methods 0.000 claims 1
- 230000000750 progressive effect Effects 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 210000000463 red nucleus Anatomy 0.000 claims 1
- 206010039073 rheumatoid arthritis Diseases 0.000 claims 1
- 208000002320 spinal muscular atrophy Diseases 0.000 claims 1
- 208000011555 sporadic pheochromocytoma Diseases 0.000 claims 1
- 208000011580 syndromic disease Diseases 0.000 claims 1
- 201000000596 systemic lupus erythematosus Diseases 0.000 claims 1
- 230000008685 targeting Effects 0.000 claims 1
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 1
- 210000001519 tissue Anatomy 0.000 claims 1
- 208000018724 torsion dystonia Diseases 0.000 claims 1
- 230000000007 visual effect Effects 0.000 claims 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US30246910P | 2010-02-08 | 2010-02-08 | |
| US61/302,469 | 2010-02-08 | ||
| US37163510P | 2010-08-06 | 2010-08-06 | |
| US61/371,635 | 2010-08-06 | ||
| PCT/US2011/024103 WO2011097643A1 (en) | 2010-02-08 | 2011-02-08 | Selective reduction of allelic variants |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016078592A Division JP6316334B2 (ja) | 2010-02-08 | 2016-04-11 | 対立遺伝子多様体の選択的低減 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2013518604A JP2013518604A (ja) | 2013-05-23 |
| JP2013518604A5 true JP2013518604A5 (https=) | 2014-03-27 |
| JP6006120B2 JP6006120B2 (ja) | 2016-10-12 |
Family
ID=44355846
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012552932A Active JP6006120B2 (ja) | 2010-02-08 | 2011-02-08 | 対立遺伝子多様体の選択的低減 |
| JP2016078592A Active JP6316334B2 (ja) | 2010-02-08 | 2016-04-11 | 対立遺伝子多様体の選択的低減 |
| JP2018055560A Active JP6652984B2 (ja) | 2010-02-08 | 2018-03-23 | 対立遺伝子多様体の選択的低減 |
| JP2020009595A Pending JP2020089382A (ja) | 2010-02-08 | 2020-01-24 | 対立遺伝子多様体の選択的低減 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016078592A Active JP6316334B2 (ja) | 2010-02-08 | 2016-04-11 | 対立遺伝子多様体の選択的低減 |
| JP2018055560A Active JP6652984B2 (ja) | 2010-02-08 | 2018-03-23 | 対立遺伝子多様体の選択的低減 |
| JP2020009595A Pending JP2020089382A (ja) | 2010-02-08 | 2020-01-24 | 対立遺伝子多様体の選択的低減 |
Country Status (9)
| Country | Link |
|---|---|
| US (5) | US8957040B2 (https=) |
| EP (3) | EP2534248B1 (https=) |
| JP (4) | JP6006120B2 (https=) |
| AU (3) | AU2011213562B2 (https=) |
| CA (1) | CA2789005A1 (https=) |
| DK (1) | DK2534248T3 (https=) |
| ES (1) | ES2733708T3 (https=) |
| IL (2) | IL221272A (https=) |
| WO (1) | WO2011097643A1 (https=) |
Families Citing this family (68)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN102282155B (zh) | 2008-12-02 | 2017-06-09 | 日本波涛生命科学公司 | 磷原子修饰的核酸的合成方法 |
| RU2612521C2 (ru) | 2009-07-06 | 2017-03-09 | Онтории, Инк. | Новые пролекарства нуклеиновых кислот и способы их применения |
| US8957040B2 (en) | 2010-02-08 | 2015-02-17 | Isis Pharmaceuticals, Inc. | Selective reduction of allelic variants |
| JP5868324B2 (ja) | 2010-09-24 | 2016-02-24 | 株式会社Wave Life Sciences Japan | 不斉補助基 |
| EP2673361B1 (en) | 2011-02-08 | 2016-04-13 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
| CN103796657B (zh) | 2011-07-19 | 2017-07-11 | 波涛生命科学有限公司 | 合成官能化核酸的方法 |
| US10202599B2 (en) * | 2011-08-11 | 2019-02-12 | Ionis Pharmaceuticals, Inc. | Selective antisense compounds and uses thereof |
| US20150315595A1 (en) * | 2012-03-12 | 2015-11-05 | Santaris Pharma A/S | Compositions and Methods for Modulation of ATXN3 Expression |
| EP2831232A4 (en) * | 2012-03-30 | 2015-11-04 | Univ Washington | METHOD FOR MODULATING TAU EXPRESSION TO REDUCE PROBLEMS AND MODIFY A NEURODEGENERATIVE SYNDROME |
| WO2013159108A2 (en) | 2012-04-20 | 2013-10-24 | Isis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
| PL2872485T3 (pl) | 2012-07-13 | 2021-05-31 | Wave Life Sciences Ltd. | Asymetryczna grupa pomocnicza |
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| US9695418B2 (en) | 2012-10-11 | 2017-07-04 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleosides and uses thereof |
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| US9909128B2 (en) * | 2012-11-15 | 2018-03-06 | The Regents Of The University Of California | Splice modulating oligonucleotides that inhibit cancer |
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| CN105264091B (zh) * | 2013-03-14 | 2020-02-28 | Ionis制药公司 | 用于调节tau表达的组合物和方法 |
| TW202246503A (zh) | 2013-07-19 | 2022-12-01 | 美商百健Ｍａ公司 | 用於調節τ蛋白表現之組合物 |
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| US10533172B2 (en) * | 2014-09-18 | 2020-01-14 | The University Of British Columbia | Allele-specific therapy for huntington disease haplotypes |
| AU2015327836B2 (en) | 2014-10-03 | 2021-07-01 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
| US20160319278A1 (en) | 2015-04-03 | 2016-11-03 | University Of Massachusetts | Fully stabilized asymmetric sirna |
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