KR20240013202A - 안티센스 핵산 - Google Patents
안티센스 핵산 Download PDFInfo
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- KR20240013202A KR20240013202A KR1020237044656A KR20237044656A KR20240013202A KR 20240013202 A KR20240013202 A KR 20240013202A KR 1020237044656 A KR1020237044656 A KR 1020237044656A KR 20237044656 A KR20237044656 A KR 20237044656A KR 20240013202 A KR20240013202 A KR 20240013202A
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- oligonucleotide
- acceptable salt
- pharmaceutically acceptable
- base sequence
- seq
- Prior art date
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- 239000003643 water by type Substances 0.000 description 1
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- A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
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- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/31—Chemical structure of the backbone
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3233—Morpholino-type ring
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- C12N2320/33—Alteration of splicing
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- C12Y—ENZYMES
- C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
- C12Y306/04—Hydrolases acting on acid anhydrides (3.6) acting on acid anhydrides; involved in cellular and subcellular movement (3.6.4)
- C12Y306/04012—DNA helicase (3.6.4.12)
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- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
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- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
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- Wood Science & Technology (AREA)
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- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
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- Animal Behavior & Ethology (AREA)
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- Plant Pathology (AREA)
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- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Virology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Enzymes And Modification Thereof (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
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| JP2021090359 | 2021-05-28 | ||
| JPJP-P-2021-090359 | 2021-05-28 | ||
| PCT/JP2022/021818 WO2022250155A1 (ja) | 2021-05-28 | 2022-05-27 | アンチセンス核酸 |
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| JP (1) | JPWO2022250155A1 (https=) |
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| CA (1) | CA3221758A1 (https=) |
| MX (1) | MX2023014092A (https=) |
| TW (1) | TW202313975A (https=) |
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| EP4342498A4 (en) * | 2021-05-13 | 2025-01-08 | National University Corporation Chiba University | ANTISENSE OLIGOMER |
Citations (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO1991009033A1 (en) | 1989-12-20 | 1991-06-27 | Anti-Gene Development Group | Uncharged morpholino-based polymers having phosphorous-containing chiral intersubunit linkages |
| WO2009064471A1 (en) | 2007-11-15 | 2009-05-22 | Avi Biopharma, Inc. | Method of synthesis of morpholino oligomers |
| WO2011052436A1 (ja) | 2009-10-29 | 2011-05-05 | 国立大学法人大阪大学 | 架橋型人工ヌクレオシドおよびヌクレオチド |
| WO2014046212A1 (ja) | 2012-09-21 | 2014-03-27 | 国立大学法人大阪大学 | グアニジン架橋を有する人工ヌクレオシドおよびオリゴヌクレオチド |
| WO2015125783A1 (ja) | 2014-02-18 | 2015-08-27 | 国立大学法人大阪大学 | 架橋型ヌクレオシドおよびヌクレオチド |
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| CN112574988A (zh) * | 2008-10-24 | 2021-03-30 | 萨雷普塔治疗公司 | 用于dmd的多外显子跳跃组合物 |
-
2022
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Patent Citations (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO1991009033A1 (en) | 1989-12-20 | 1991-06-27 | Anti-Gene Development Group | Uncharged morpholino-based polymers having phosphorous-containing chiral intersubunit linkages |
| WO2009064471A1 (en) | 2007-11-15 | 2009-05-22 | Avi Biopharma, Inc. | Method of synthesis of morpholino oligomers |
| WO2011052436A1 (ja) | 2009-10-29 | 2011-05-05 | 国立大学法人大阪大学 | 架橋型人工ヌクレオシドおよびヌクレオチド |
| WO2014046212A1 (ja) | 2012-09-21 | 2014-03-27 | 国立大学法人大阪大学 | グアニジン架橋を有する人工ヌクレオシドおよびオリゴヌクレオチド |
| WO2015125783A1 (ja) | 2014-02-18 | 2015-08-27 | 国立大学法人大阪大学 | 架橋型ヌクレオシドおよびヌクレオチド |
Non-Patent Citations (8)
| Title |
|---|
| Am. J. Hum. Genet.(1997) 60: 330-341 |
| Annu. Rev. Pharmacol. Toxicol.(2010) 50: 259-293 |
| Geriatr Gerontol Int(2013) 13: 475-481 |
| Human Genetics(1981) 58: 310-316 |
| J.Am.Chem.Soc(2016) 138: 15663-15672 |
| J.Med.Chem.(2016) 59: 9645-9667 |
| Nature(1992) 355: 735-738 |
| 피부임상(2000) 40: 1512-1513 |
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| JPWO2022250155A1 (https=) | 2022-12-01 |
| AU2022279820A1 (en) | 2023-12-14 |
| EP4349987A1 (en) | 2024-04-10 |
| CN117500925A (zh) | 2024-02-02 |
| CA3221758A1 (en) | 2022-12-01 |
| WO2022250155A1 (ja) | 2022-12-01 |
| MX2023014092A (es) | 2023-12-11 |
| AU2022279820A9 (en) | 2024-01-04 |
| TW202313975A (zh) | 2023-04-01 |
| US20250084411A1 (en) | 2025-03-13 |
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