TW202117015A - 使DUX4 pre-mRNA之剪接變化之反義寡核苷酸 - Google Patents
使DUX4 pre-mRNA之剪接變化之反義寡核苷酸 Download PDFInfo
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- TW202117015A TW202117015A TW109123473A TW109123473A TW202117015A TW 202117015 A TW202117015 A TW 202117015A TW 109123473 A TW109123473 A TW 109123473A TW 109123473 A TW109123473 A TW 109123473A TW 202117015 A TW202117015 A TW 202117015A
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- dux4
- oligonucleotide
- pharmaceutically acceptable
- acceptable salt
- sequence
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- 239000004094 surface-active agent Substances 0.000 description 1
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- 235000020357 syrup Nutrition 0.000 description 1
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- 229940095064 tartrate Drugs 0.000 description 1
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- RTKIYNMVFMVABJ-UHFFFAOYSA-L thimerosal Chemical compound [Na+].CC[Hg]SC1=CC=CC=C1C([O-])=O RTKIYNMVFMVABJ-UHFFFAOYSA-L 0.000 description 1
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- ITMCEJHCFYSIIV-UHFFFAOYSA-M triflate Chemical compound [O-]S(=O)(=O)C(F)(F)F ITMCEJHCFYSIIV-UHFFFAOYSA-M 0.000 description 1
- RYFMWSXOAZQYPI-UHFFFAOYSA-K trisodium phosphate Chemical compound [Na+].[Na+].[Na+].[O-]P([O-])([O-])=O RYFMWSXOAZQYPI-UHFFFAOYSA-K 0.000 description 1
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- 229940046009 vitamin E Drugs 0.000 description 1
- 239000011709 vitamin E Substances 0.000 description 1
- 239000001993 wax Substances 0.000 description 1
- 150000003751 zinc Chemical class 0.000 description 1
Images
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| US6096881A (en) | 1997-05-30 | 2000-08-01 | Hybridon, Inc. | Sulfur transfer reagents for oligonucleotide synthesis |
| JP4236812B2 (ja) | 1997-09-12 | 2009-03-11 | エクシコン エ/エス | オリゴヌクレオチド類似体 |
| ES2234563T5 (es) | 1999-02-12 | 2018-01-17 | Daiichi Sankyo Company, Limited | Nuevos análogos de nucleósidos y oligonucleótidos |
| US6261840B1 (en) | 2000-01-18 | 2001-07-17 | Isis Pharmaceuticals, Inc. | Antisense modulation of PTP1B expression |
| ES2596977T3 (es) * | 2010-09-02 | 2017-01-13 | Université de Mons | Agentes útiles en el tratamiento de la distrofia muscular facioescapulohumeral |
| EP2736539B1 (en) * | 2011-07-25 | 2017-08-23 | Nationwide Children's Hospital, Inc. | Recombinant virus products and methods for inhibition of expression of dux4 |
| WO2013120038A2 (en) * | 2012-02-10 | 2013-08-15 | Regents Of The University Of Minnesota | Morpholino targeting dux4 for treating fshd |
| WO2014109384A1 (ja) | 2013-01-10 | 2014-07-17 | 塩野義製薬株式会社 | 架橋型核酸誘導体の製造方法 |
| US20180237775A1 (en) * | 2015-09-21 | 2018-08-23 | Association Institut De Myologie | Antisense oligonucleotides and uses thereof |
| JP6966463B2 (ja) * | 2016-02-26 | 2021-11-17 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | 組換えウイルス産物及びdux4エクソンスキッピングを誘導するための方法 |
| CN109462995A (zh) | 2016-05-04 | 2019-03-12 | 波涛生命科学有限公司 | 生物活性剂的方法和组合物 |
| WO2019060432A2 (en) * | 2017-09-19 | 2019-03-28 | Children's National Medical Center | GAPMERS AND METHODS OF USING THE SAME FOR THE TREATMENT OF MUSCLE DYSTROPHY |
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| JP7627495B2 (ja) | 2025-02-06 |
| JPWO2021010301A1 (https=) | 2021-01-21 |
| BR112021024764A2 (pt) | 2022-04-19 |
| WO2021010301A1 (ja) | 2021-01-21 |
| IL288596A (en) | 2022-02-01 |
| KR20220032004A (ko) | 2022-03-15 |
| AU2020313255A1 (en) | 2022-01-06 |
| US20220364086A1 (en) | 2022-11-17 |
| EP3998108A4 (en) | 2024-10-30 |
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