JPWO2023214512A1 - - Google Patents

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Publication number
JPWO2023214512A1
JPWO2023214512A1 JP2024519192A JP2024519192A JPWO2023214512A1 JP WO2023214512 A1 JPWO2023214512 A1 JP WO2023214512A1 JP 2024519192 A JP2024519192 A JP 2024519192A JP 2024519192 A JP2024519192 A JP 2024519192A JP WO2023214512 A1 JPWO2023214512 A1 JP WO2023214512A1
Authority
JP
Japan
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2024519192A
Other languages
Japanese (ja)
Other versions
JPWO2023214512A5 (https=
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed filed Critical
Publication of JPWO2023214512A1 publication Critical patent/JPWO2023214512A1/ja
Publication of JPWO2023214512A5 publication Critical patent/JPWO2023214512A5/ja
Pending legal-status Critical Current

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12PFERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P19/00Preparation of compounds containing saccharide radicals
    • C12P19/26Preparation of nitrogen-containing carbohydrates
    • C12P19/28N-glycosides
    • C12P19/30Nucleotides
    • C12P19/34Polynucleotides, e.g. nucleic acids, oligoribonucleotides
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
JP2024519192A 2022-05-06 2023-04-20 Pending JPWO2023214512A1 (https=)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
JP2022076674 2022-05-06
PCT/JP2023/015828 WO2023214512A1 (ja) 2022-05-06 2023-04-20 人工rna分子

Publications (2)

Publication Number Publication Date
JPWO2023214512A1 true JPWO2023214512A1 (https=) 2023-11-09
JPWO2023214512A5 JPWO2023214512A5 (https=) 2025-01-20

Family

ID=88646433

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024519192A Pending JPWO2023214512A1 (https=) 2022-05-06 2023-04-20

Country Status (4)

Country Link
US (1) US20250297253A1 (https=)
EP (1) EP4520825A1 (https=)
JP (1) JPWO2023214512A1 (https=)
WO (1) WO2023214512A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025217551A1 (en) * 2024-04-12 2025-10-16 University Of Miami Microrna-mediated obstruction of stem-loop alternative splicing

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2013521791A (ja) * 2010-03-17 2013-06-13 アソシアシオン インスティテュト ドゥ ミョロジー 神経筋疾患の治療のための修飾型U7snRNA
JP2019519218A (ja) * 2016-05-18 2019-07-11 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. 調節性ポリヌクレオチド
WO2021117729A1 (ja) * 2019-12-09 2021-06-17 アステラス製薬株式会社 標的rnaを編集するための機能性領域を付加したアンチセンス型ガイドrna

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
MX2018007987A (es) 2015-12-28 2019-01-10 Novartis Ag Composiciones y metodos para el tratamiento de hemoglobinopatias.

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2013521791A (ja) * 2010-03-17 2013-06-13 アソシアシオン インスティテュト ドゥ ミョロジー 神経筋疾患の治療のための修飾型U7snRNA
JP2019519218A (ja) * 2016-05-18 2019-07-11 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. 調節性ポリヌクレオチド
WO2021117729A1 (ja) * 2019-12-09 2021-06-17 アステラス製薬株式会社 標的rnaを編集するための機能性領域を付加したアンチセンス型ガイドrna

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
FUMIO HARADA ET AL.: "Nucleotide sequences of 4.5S RNAs associated with poly (A) -containing RNAs of mouse and hamster cel", NUCLEIC ACIDS RESEARCH, vol. 8, no. 6, JPN6025038760, 1980, pages 1273 - 1286, ISSN: 0005813626 *
ISHIDA, KENTARO ET AL.: "Regulation of gene expression via retrotransposon insertions and the noncoding RNA 4.5S RNAH", GENES TO CELLS, vol. 20, no. 11, JPN6023028287, 3 September 2015 (2015-09-03), pages 887 - 901, ISSN: 0005698322 *

Also Published As

Publication number Publication date
EP4520825A1 (en) 2025-03-12
WO2023214512A1 (ja) 2023-11-09
US20250297253A1 (en) 2025-09-25

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