JP2025504661A - 偽エクソンを標的とする対立遺伝子特異的スプライススイッチングオリゴヌクレオチド - Google Patents

偽エクソンを標的とする対立遺伝子特異的スプライススイッチングオリゴヌクレオチド Download PDF

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Publication number
JP2025504661A
JP2025504661A JP2024544641A JP2024544641A JP2025504661A JP 2025504661 A JP2025504661 A JP 2025504661A JP 2024544641 A JP2024544641 A JP 2024544641A JP 2024544641 A JP2024544641 A JP 2024544641A JP 2025504661 A JP2025504661 A JP 2025504661A
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Prior art keywords
sso
pseudoexon
disease
splice site
mrna
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Pending
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JP2024544641A
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Japanese (ja)
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JP2025504661A5 (https=
Inventor
ブラゲ ストルスティン アンドレセン
トーマス コード ドクター
リセ ローレ ホルム
ウルリカ シモーネ スパングスベルグ ペーターセン
ギッテ ホフマン ブルーン
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Syddansk Universitet
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Syddansk Universitet
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Priority claimed from PCT/EP2022/051790 external-priority patent/WO2022162020A2/en
Application filed by Syddansk Universitet filed Critical Syddansk Universitet
Publication of JP2025504661A publication Critical patent/JP2025504661A/ja
Publication of JP2025504661A5 publication Critical patent/JP2025504661A5/ja
Pending legal-status Critical Current

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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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
JP2024544641A 2022-01-26 2023-01-26 偽エクソンを標的とする対立遺伝子特異的スプライススイッチングオリゴヌクレオチド Pending JP2025504661A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
EPPCT/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

Publications (2)

Publication Number Publication Date
JP2025504661A true JP2025504661A (ja) 2025-02-14
JP2025504661A5 JP2025504661A5 (https=) 2026-02-16

Family

ID=85108839

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024544641A Pending JP2025504661A (ja) 2022-01-26 2023-01-26 偽エクソンを標的とする対立遺伝子特異的スプライススイッチングオリゴヌクレオチド

Country Status (7)

Country Link
US (1) US20250101419A1 (https=)
EP (1) EP4469572A1 (https=)
JP (1) JP2025504661A (https=)
CN (1) CN119013399A (https=)
AU (1) AU2023210747A1 (https=)
CA (1) CA3247528A1 (https=)
WO (1) WO2023144263A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024243087A2 (en) * 2023-05-19 2024-11-28 Leverna Therapeutics Inc. Compositions and methods for regulating expression of atxn2

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Also Published As

Publication number Publication date
WO2023144263A1 (en) 2023-08-03
AU2023210747A1 (en) 2024-08-01
CA3247528A1 (en) 2023-08-03
CN119013399A (zh) 2024-11-22
EP4469572A1 (en) 2024-12-04
US20250101419A1 (en) 2025-03-27

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