JP7759106B2 - Ｒｎａ分子の高効率の組換えのための組成物および方法 - Google Patents

Ｒｎａ分子の高効率の組換えのための組成物および方法

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Publication number
JP7759106B2
JP7759106B2 JP2022526727A JP2022526727A JP7759106B2 JP 7759106 B2 JP7759106 B2 JP 7759106B2 JP 2022526727 A JP2022526727 A JP 2022526727A JP 2022526727 A JP2022526727 A JP 2022526727A JP 7759106 B2 JP7759106 B2 JP 7759106B2
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rna
sequence
protein
dimerization domain
molecule
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JP2023500957A (ja
JP2023500957A5 (https=
Inventor
ルーカス クリストフ バックマン，
サミュエル ローレンス パフ，
Original Assignee
ソーク インスティテュート フォー バイオロジカル スタディーズ
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Priority claimed from PCT/US2020/025430 external-priority patent/WO2020205604A1/en
Application filed by ソーク インスティテュート フォー バイオロジカル スタディーズ filed Critical ソーク インスティテュート フォー バイオロジカル スタディーズ
Publication of JP2023500957A publication Critical patent/JP2023500957A/ja
Publication of JP2023500957A5 publication Critical patent/JP2023500957A5/ja
Priority to JP2025079571A priority Critical patent/JP2025118824A/ja
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • C12N15/115—Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/64—General methods for preparing the vector, for introducing it into the cell or for selecting the vector-containing host
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • C12N2310/16—Aptamers
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor
    • C12N2840/445—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor for trans-splicing, e.g. polypyrimidine tract, branch point splicing

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Virology (AREA)
  • Cell Biology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
  • Saccharide Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2022526727A 2019-11-11 2020-09-30 Ｒｎａ分子の高効率の組換えのための組成物および方法 Active JP7759106B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025079571A JP2025118824A (ja) 2019-11-11 2025-05-12 Ｒｎａ分子の高効率の組換えのための組成物および方法

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962933714P 2019-11-11 2019-11-11
US62/933,714 2019-11-11
USPCT/US2020/025430 2020-03-27
PCT/US2020/025430 WO2020205604A1 (en) 2019-03-29 2020-03-27 High-efficiency reconstitution of rna molecules
PCT/US2020/053643 WO2021096605A1 (en) 2019-11-11 2020-09-30 Compositions and methods for high-efficiency recombination of rna molecules

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025079571A Division JP2025118824A (ja) 2019-11-11 2025-05-12 Ｒｎａ分子の高効率の組換えのための組成物および方法

Publications (3)

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JP2023500957A JP2023500957A (ja) 2023-01-11
JP2023500957A5 JP2023500957A5 (https=) 2023-10-10
JP7759106B2 true JP7759106B2 (ja) 2025-10-23

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JP2025079571A Pending JP2025118824A (ja) 2019-11-11 2025-05-12 Ｒｎａ分子の高効率の組換えのための組成物および方法

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Country Status (11)

Country Link
US (1) US20220265855A1 (https=)
EP (1) EP4058571A1 (https=)
JP (2) JP7759106B2 (https=)
KR (1) KR20220113940A (https=)
CN (1) CN114945666A (https=)
AU (1) AU2020384996A1 (https=)
BR (1) BR112022009006A2 (https=)
CA (1) CA3157799A1 (https=)
IL (1) IL292904A (https=)
MX (1) MX2022005670A (https=)
WO (1) WO2021096605A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020205604A1 (en) 2019-03-29 2020-10-08 Salk Institute For Biological Studies High-efficiency reconstitution of rna molecules
KR20220157944A (ko) 2020-02-21 2022-11-29 아카우오스, 인크. 인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법
AU2022337146A1 (en) * 2021-09-03 2024-03-14 Tacit Therapeutics, Inc. Rna editing via recruitment of spliceosome components
CA3240197A1 (en) * 2021-12-10 2025-02-26 Aavantibio, Inc. Compositions comprising kozak sequences selected for enhanced expression
WO2023250324A2 (en) * 2022-06-21 2023-12-28 The Regents Of The University Of California Compositions and methods for reducing rna levels
US20260055400A1 (en) * 2022-06-21 2026-02-26 The Regents Of The University Of California Compositions and methods for treating huntington's disease

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060134658A1 (en) 2004-08-09 2006-06-22 Garcia-Blanco Mariano A Use of RNA trans-splicing for generation of interfering RNA molecules
JP2017520255A (ja) 2014-07-03 2017-07-27 ジェネンテック， インコーポレイテッド ポリペプチド発現系

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6110743A (en) 1995-02-10 2000-08-29 The Regents Of The University Of California Development and use of human pancreatic cell lines

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060134658A1 (en) 2004-08-09 2006-06-22 Garcia-Blanco Mariano A Use of RNA trans-splicing for generation of interfering RNA molecules
JP2017520255A (ja) 2014-07-03 2017-07-27 ジェネンテック， インコーポレイテッド ポリペプチド発現系

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
Lorain, S. et al.，"Exon exchange approach to repair Duchenne dystrophin transcripts"，PLoS One，2010年，Vol. 5; e10894，pp. 1-11
Nakayama, K. et al.，"Gene transfer-mediated pre-mRNA segmental trans-splicing as a strategy to deliver intracellular toxins for cancer therapy"，Cancer Res.，2005年，Vol. 65，pp. 254-263
Pergolizzi, R. G. et al.，"Genetic medicine at the RNA level: modifications of the genetic repertoire for therapeutic purposes by pre-mRNA trans-splicing"，C R Biol.，2004年，Vol. 327，pp. 695-709
Pergolizzi, R. G. et al.，"In vivo trans-splicing of 5' and 3' segments of pre-mRNA directed by corresponding DNA sequences delivered by gene transfer"，Mol. Ther.，2003年，Vol. 8，pp. 999-1008

Also Published As

Publication number Publication date
JP2025118824A (ja) 2025-08-13
JP2023500957A (ja) 2023-01-11
CN114945666A (zh) 2022-08-26
MX2022005670A (es) 2022-07-19
WO2021096605A1 (en) 2021-05-20
US20220265855A1 (en) 2022-08-25
IL292904A (en) 2022-07-01
EP4058571A1 (en) 2022-09-21
KR20220113940A (ko) 2022-08-17
AU2020384996A1 (en) 2022-06-23
BR112022009006A2 (pt) 2022-10-11
CA3157799A1 (en) 2021-05-20

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