AU2023210747A1 - Allele specific splice switching oligonucleotides targeting pseudoexons - Google Patents
Allele specific splice switching oligonucleotides targeting pseudoexons Download PDFInfo
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- AU2023210747A1 AU2023210747A1 AU2023210747A AU2023210747A AU2023210747A1 AU 2023210747 A1 AU2023210747 A1 AU 2023210747A1 AU 2023210747 A AU2023210747 A AU 2023210747A AU 2023210747 A AU2023210747 A AU 2023210747A AU 2023210747 A1 AU2023210747 A1 AU 2023210747A1
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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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/11—Antisense
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| AUPCT/EP2022/051790 | 2022-01-26 | ||
| PCT/EP2022/051790 WO2022162020A2 (en) | 2021-01-26 | 2022-01-26 | Splice switching oligonucleotides targeting pseudoexons |
| DKPA202270346 | 2022-06-29 | ||
| DKPA202270346 | 2022-06-29 | ||
| PCT/EP2023/051916 WO2023144263A1 (en) | 2022-01-26 | 2023-01-26 | Allele specific splice switching oligonucleotides targeting pseudoexons |
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| CN108603230A (zh) * | 2015-10-09 | 2018-09-28 | 南安普敦大学 | 基因表达的调节与蛋白质表达失调的筛选 |
| WO2017087282A1 (en) * | 2015-11-18 | 2017-05-26 | Rosalind Franklin University Of Medicine And Science | Antisense compounds targeting leucine-rich repeat kinase 2 (lrrk2) for the treatment of parkinsons disease |
| KR102318434B1 (ko) * | 2017-08-25 | 2021-11-01 | 스톡 테라퓨틱스, 인크. | 병태 및 질환 치료용 안티센스 올리고머 |
| TWI833770B (zh) * | 2018-06-27 | 2024-03-01 | 美商Ionis製藥公司 | 用於減少 lrrk2 表現之化合物及方法 |
| US12595479B2 (en) * | 2019-03-20 | 2026-04-07 | President And Fellows Of Harvard College | Antisense oligonucleotide-based progranulin augmentation therapy in neurodegenerative diseases |
| WO2022119881A1 (en) * | 2020-12-01 | 2022-06-09 | Emendobio Inc. | Differential knockout of a heterozygous allele of lrrk2 |
| CA3204779A1 (en) * | 2021-01-26 | 2022-08-04 | Brage Storstein Andresen | Splice switching oligonucleotides targeting pseudoexons |
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| WO2023144263A1 (en) | 2023-08-03 |
| CA3247528A1 (en) | 2023-08-03 |
| CN119013399A (zh) | 2024-11-22 |
| EP4469572A1 (en) | 2024-12-04 |
| JP2025504661A (ja) | 2025-02-14 |
| US20250101419A1 (en) | 2025-03-27 |
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