CN117431244A - 用于调节ube3a-ats的化合物和方法 - Google Patents
用于调节ube3a-ats的化合物和方法 Download PDFInfo
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- CN117431244A CN117431244A CN202311298093.6A CN202311298093A CN117431244A CN 117431244 A CN117431244 A CN 117431244A CN 202311298093 A CN202311298093 A CN 202311298093A CN 117431244 A CN117431244 A CN 117431244A
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Classifications
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- 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
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- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
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- C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
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- C12N2310/315—Phosphorothioates
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- C12N2310/321—2'-O-R Modification
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- C12N2310/323—Chemical structure of the sugar modified ring structure
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- C12N2310/3341—5-Methylcytosine
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| US201962826521P | 2019-03-29 | 2019-03-29 | |
| US62/826,521 | 2019-03-29 | ||
| US201962877765P | 2019-07-23 | 2019-07-23 | |
| US62/877,765 | 2019-07-23 | ||
| CN202080025171.3A CN113728104B (zh) | 2019-03-29 | 2020-03-27 | 用于调节ube3a-ats的化合物和方法 |
| PCT/US2020/025110 WO2020205463A1 (en) | 2019-03-29 | 2020-03-27 | Compounds and methods for modulating ube3a-ats |
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| Publication number | Priority date | Publication date | Assignee | Title |
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| WO2014004572A2 (en) | 2012-06-25 | 2014-01-03 | Isis Pharmaceuticals, Inc. | Modulation of ube3a-ats expression |
| LT3374509T (lt) | 2015-11-12 | 2021-03-10 | F. Hoffmann-La Roche Ag | Oligonukleotidai, skirti sukelti iš tėvo paveldėto ube3a raišką |
| PT3947684T (pt) | 2019-03-29 | 2025-05-19 | Ionis Pharmaceuticals Inc | Compostos e métodos para modular ube3a‑ats |
| US20220259601A1 (en) * | 2021-02-17 | 2022-08-18 | Q-State Biosciences, Inc. | Ube3a antisense therapeutics |
| WO2025100980A1 (ko) * | 2023-11-08 | 2025-05-15 | 한국과학기술원 | Ube3a를 표적으로 하여 발현증강을 유도하는 안티센스 올리고뉴클레오티드 및 이의 용도 |
| WO2025101991A1 (en) * | 2023-11-09 | 2025-05-15 | Ionis Pharmaceuticals, Inc. | Methods for modulating ube3a-ats |
| WO2025240846A1 (en) * | 2024-05-17 | 2025-11-20 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating apoe expression |
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