CN114945666A - 用于rna分子高效重组的组合物和方法 - Google Patents

用于rna分子高效重组的组合物和方法 Download PDF

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Publication number
CN114945666A
CN114945666A CN202080092659.8A CN202080092659A CN114945666A CN 114945666 A CN114945666 A CN 114945666A CN 202080092659 A CN202080092659 A CN 202080092659A CN 114945666 A CN114945666 A CN 114945666A
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rna
sequence
protein
composition
dimerization domain
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CN202080092659.8A
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Chinese (zh)
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L·C·巴赫曼
S·L·普法夫
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Salk Institute for Biological Studies
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Salk Institute for Biological Studies
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Priority claimed from PCT/US2020/025430 external-priority patent/WO2020205604A1/en
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Publication of CN114945666A publication Critical patent/CN114945666A/zh
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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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • 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
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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
    • C12N15/115—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
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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
    • C12N15/63—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
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    • 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
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    • 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

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  • 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)
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  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
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  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
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  • 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)
CN202080092659.8A 2019-11-11 2020-09-30 用于rna分子高效重组的组合物和方法 Pending CN114945666A (zh)

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

Publications (1)

Publication Number Publication Date
CN114945666A true CN114945666A (zh) 2022-08-26

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ID=75911442

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CN202080092659.8A Pending CN114945666A (zh) 2019-11-11 2020-09-30 用于rna分子高效重组的组合物和方法

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

Family Cites Families (3)

* 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
US20060134658A1 (en) * 2004-08-09 2006-06-22 Garcia-Blanco Mariano A Use of RNA trans-splicing for generation of interfering RNA molecules
ES2763526T3 (es) * 2014-07-03 2020-05-29 Hoffmann La Roche Sistemas de expresión de polipéptidos

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Publication number Publication date
JP2025118824A (ja) 2025-08-13
JP2023500957A (ja) 2023-01-11
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
JP7759106B2 (ja) 2025-10-23
AU2020384996A1 (en) 2022-06-23
BR112022009006A2 (pt) 2022-10-11
CA3157799A1 (en) 2021-05-20

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