AU2020290790B2 - Targeted gene editing constructs and methods of using the same - Google Patents

Targeted gene editing constructs and methods of using the same

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Publication number
AU2020290790B2
AU2020290790B2 AU2020290790A AU2020290790A AU2020290790B2 AU 2020290790 B2 AU2020290790 B2 AU 2020290790B2 AU 2020290790 A AU2020290790 A AU 2020290790A AU 2020290790 A AU2020290790 A AU 2020290790A AU 2020290790 B2 AU2020290790 B2 AU 2020290790B2
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nucleic acid
amino acid
sequence
modified
seq
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AU2020290790A1 (en
Inventor
Marc GÜELL CARGOL
Dimitrie IVANCIC DJERMANOVIC
Maria PALLARÈS MASMITJA
Avencia SÁNCHEZ-MEJÍAS GARCÍA
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Universitat Pompeu Fabra
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Universitat Pompeu Fabra
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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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
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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
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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
    • C12N15/62—DNA sequences coding for fusion proteins
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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
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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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
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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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
    • C12N15/79—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
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241—Nucleotidyltransferases (2.7.7)
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16022—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—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
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian
    • C—CHEMISTRY; METALLURGY
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/90—Vectors containing a transposable element

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  • Health & Medical Sciences (AREA)
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  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Immunology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)
AU2020290790A 2019-06-11 2020-06-11 Targeted gene editing constructs and methods of using the same Active AU2020290790B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962860186P 2019-06-11 2019-06-11
US62/860,186 2019-06-11
PCT/IB2020/055507 WO2020250181A1 (en) 2019-06-11 2020-06-11 Targeted gene editing constructs and methods of using the same

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AU2020290790A1 AU2020290790A1 (en) 2022-01-27
AU2020290790B2 true AU2020290790B2 (en) 2026-02-19

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US (1) US20220235379A1 (enExample)
EP (1) EP3983541A1 (enExample)
JP (1) JP7830129B2 (enExample)
KR (1) KR102990253B1 (enExample)
CN (1) CN114026240A (enExample)
AU (1) AU2020290790B2 (enExample)
BR (1) BR112021024828A2 (enExample)
CA (1) CA3141422A1 (enExample)
IL (1) IL288794A (enExample)
MX (1) MX2021015157A (enExample)
WO (1) WO2020250181A1 (enExample)

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JP7419168B2 (ja) * 2020-06-10 2024-01-22 株式会社東芝 改変型ｐｉｇｇｙＢａｃトランスポゼースのポリペプチド、それをコードするポリヌクレオチド、導入キャリア、キット、細胞のゲノムに目的配列を組込む方法及び細胞製造方法
WO2022129430A1 (en) * 2020-12-16 2022-06-23 Universitat Pompeu Fabra Therapeutic lama2 payload for treatment of congenital muscular dystrophy
AU2021403660A1 (en) 2020-12-16 2023-07-06 Universitat Pompeu Fabra Programmable transposases and uses thereof
AU2022272723A1 (en) * 2021-05-14 2023-11-30 Becton, Dickinson And Company Methods for making libraries for nucleic acid sequencing
AU2022273536A1 (en) * 2021-05-14 2023-11-16 Becton, Dickinson And Company Multiplexed unbiased nucleic acid amplification method
FR3124522B1 (fr) * 2021-06-25 2025-02-28 Cherbonneau Francois Composition et méthode permettant l’édition génomique
WO2023060089A2 (en) * 2021-10-04 2023-04-13 Poseida Therapeutics, Inc. Transposases and uses thereof
AU2022425534A1 (en) * 2021-12-30 2024-07-04 Regel Therapeutics, Inc. Compositions for modulating expression of sodium voltage-gated channel alpha subunit 1 and uses thereof
WO2023141504A2 (en) * 2022-01-19 2023-07-27 Genomeminer, Inc. (Formally Tupac Bio, Inc.) Dcas9-integrase for targeted genome editing
US20250158010A1 (en) 2022-02-15 2025-05-15 Lg Energy Solution, Ltd. Apparatus for manufacturing secondary battery and method for manufacturing secondary battery using the same
CN116218904B (zh) * 2022-11-03 2025-04-15 国药中生生物技术研究院有限公司 一种可提高工程化细胞外泌体中特定核酸分子载量的方法及其应用
CN118185901B (zh) 2022-12-12 2025-03-21 北京精缮生物科技有限责任公司 一种PBase蛋白、融合蛋白、核酸和基因整合系统及应用
EP4504923A4 (en) * 2023-03-27 2025-12-31 Beijing Astragenomics Tech Co Ltd ISOLATED TRANSPOSASE AND ITS USE
WO2024227131A1 (en) * 2023-04-27 2024-10-31 Rensselaer Polytechnic Institute Recombinant enzyme for the accurate insertion of dna sequences in eukaryotic cells
WO2024246338A2 (en) 2023-06-01 2024-12-05 Integra Therapeutics Novel transposases and uses thereof
WO2025212757A2 (en) * 2024-04-03 2025-10-09 Buck Institute For Research On Aging Cas enabled targeted transposition
WO2026087488A1 (en) 2024-10-21 2026-04-30 Integra Therapeutics Gene editing tools and compositions for cell engineering
CN119842638A (zh) * 2025-01-20 2025-04-18 天津大学 融合酶突变体及其在氟代白藜芦醇合成中的应用

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JP7830129B2 (ja) 2026-03-16
WO2020250181A1 (en) 2020-12-17
KR102990253B1 (ko) 2026-07-10
MX2021015157A (es) 2022-03-17
AU2020290790A1 (en) 2022-01-27
JP2022540318A (ja) 2022-09-15
CN114026240A (zh) 2022-02-08
BR112021024828A2 (pt) 2022-01-25
EP3983541A1 (en) 2022-04-20
CA3141422A1 (en) 2020-12-17
KR20220019794A (ko) 2022-02-17
IL288794A (en) 2022-02-01
US20220235379A1 (en) 2022-07-28

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