UA128437C2 - Сполуки і способи модулювання ube3a-ats - Google Patents
Сполуки і способи модулювання ube3a-ats Download PDFInfo
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- UA128437C2 UA128437C2 UAA202106053A UAA202106053A UA128437C2 UA 128437 C2 UA128437 C2 UA 128437C2 UA A202106053 A UAA202106053 A UA A202106053A UA A202106053 A UAA202106053 A UA A202106053A UA 128437 C2 UA128437 C2 UA 128437C2
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- certain embodiments
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Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- 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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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
- C12N2310/111—Antisense spanning the whole gene, or a large part of it
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/11—Antisense
- C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
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| Application Number | Priority Date | Filing Date | Title |
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| US201962826521P | 2019-03-29 | 2019-03-29 | |
| US201962877765P | 2019-07-23 | 2019-07-23 | |
| PCT/US2020/025110 WO2020205463A1 (en) | 2019-03-29 | 2020-03-27 | Compounds and methods for modulating ube3a-ats |
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| UAA202106053A UA128437C2 (uk) | 2019-03-29 | 2020-03-27 | Сполуки і способи модулювання ube3a-ats |
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| Publication number | Priority date | Publication date | Assignee | Title |
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| ES2809199T3 (es) | 2012-06-25 | 2021-03-03 | Ionis Pharmaceuticals Inc | Modulación de la expresión de UBE3A-ATS |
| HUE067905T2 (hu) | 2015-11-12 | 2024-11-28 | Hoffmann La Roche | Oligonukleotidok paternális UBE3A expresszió indukálására |
| FI3947684T3 (fi) | 2019-03-29 | 2025-05-27 | Ionis Pharmaceuticals Inc | Yhdisteitä ja menetelmiä ube3a-ats:n moduloimiseksi |
| CA3211396A1 (en) * | 2021-02-17 | 2022-08-25 | Q-State Biosciences, Inc. | Ube3a antisense therapeutics |
| KR20250068540A (ko) * | 2023-11-08 | 2025-05-16 | 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 |
| US20250353873A1 (en) * | 2024-05-17 | 2025-11-20 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating apoe expression |
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