BR112021024828A2 - Construtos de edição de gene alvejado e métodos de uso dos mesmos - Google Patents

Construtos de edição de gene alvejado e métodos de uso dos mesmos

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Publication number
BR112021024828A2
BR112021024828A2 BR112021024828A BR112021024828A BR112021024828A2 BR 112021024828 A2 BR112021024828 A2 BR 112021024828A2 BR 112021024828 A BR112021024828 A BR 112021024828A BR 112021024828 A BR112021024828 A BR 112021024828A BR 112021024828 A2 BR112021024828 A2 BR 112021024828A2
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BR
Brazil
Prior art keywords
nucleic acid
methods
encodes
gene editing
targeted gene
Prior art date
Application number
BR112021024828A
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English (en)
Portuguese (pt)
Inventor
Sánchez-Mejías García Avencia
Ivancic Djermanovic Dimitrie
Güell Cargol Marc
Pallarès Masmitja Maria
Original Assignee
Univ Pompeu Fabra
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Publication date
Application filed by Univ Pompeu Fabra filed Critical Univ Pompeu Fabra
Publication of BR112021024828A2 publication Critical patent/BR112021024828A2/pt

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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
    • C12N15/87—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
    • 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
    • C12N15/10—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
    • C12N15/113—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
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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
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    • 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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    • C12N15/86—Viral vectors
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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)
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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/90—Vectors containing a transposable element

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  • Bioinformatics & Cheminformatics (AREA)
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  • Wood Science & Technology (AREA)
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  • Molecular Biology (AREA)
  • Biochemistry (AREA)
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  • Physics & Mathematics (AREA)
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  • Cell Biology (AREA)
  • Mycology (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)
BR112021024828A 2019-06-11 2020-06-11 Construtos de edição de gene alvejado e métodos de uso dos mesmos BR112021024828A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962860186P 2019-06-11 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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BR112021024828A2 true BR112021024828A2 (pt) 2022-01-25

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US (1) US20220235379A1 (enExample)
EP (1) EP3983541A1 (enExample)
JP (1) JP7830129B2 (enExample)
KR (1) KR20220019794A (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
JP2023554504A (ja) 2020-12-16 2023-12-27 ユニバーシタット ポンペウ ファブラ プログラム可能なトランスポザーゼおよびその使用
CA3202411A1 (en) * 2020-12-16 2022-06-23 Marc Guell Cargol Therapeutic lama2 payload for treatment of congenital muscular dystrophy
CA3217426A1 (en) * 2021-05-14 2022-11-17 Alvaro GODINEZ Multiplexed unbiased nucleic acid amplification method
KR20240099249A (ko) * 2021-10-04 2024-06-28 포세이다 테라퓨틱스, 인크. 트랜스포사제 및 이의 용도
CA3244955A1 (en) * 2021-12-30 2023-07-06 Regel Therapeutics, Inc. Compositions for modulating the expression of the alpha 1 subunit of the voltage-gated sodium channel and their uses
WO2023141504A2 (en) * 2022-01-19 2023-07-27 Genomeminer, Inc. (Formally Tupac Bio, Inc.) Dcas9-integrase for targeted genome editing
KR20230122932A (ko) 2022-02-15 2023-08-22 주식회사 엘지에너지솔루션 이차전지 제조장치 및 이를 이용하는 이차전지 제조방법
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
CN120574802A (zh) * 2023-03-27 2025-09-02 北京星辰集因科技有限责任公司 一种分离的转座酶及其用途
CN121712899A (zh) * 2023-04-27 2026-03-20 伦斯勒理工学院 用于在真核细胞中准确插入dna序列的重组酶
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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SI1546322T1 (sl) * 2002-07-24 2011-05-31 Manoa Biosciences Inc Vektorji na osnovi transpozona in metode integracije nukleinskih kislin
US9783790B2 (en) * 2011-07-15 2017-10-10 The Johns Hopkins University Trichoplusia ni piggybac transposases with reduced integration activity
WO2015157611A2 (en) * 2014-04-11 2015-10-15 The Johns Hopkins University Improvements to eukaryotic transposase mutants and transposon end compositions for modifying nucleic acids and methods for production and use in the generation of sequencing libraries
EP3277805B1 (en) * 2015-03-31 2025-05-07 SOHM, Inc. Cas 9 retroviral integrase systems for targeted incorporation of a dna sequence into a genome of a cell
AU2016373365B2 (en) * 2015-12-14 2021-07-08 GenomeFrontier Therapeutics TW Co., Ltd. Transposon system, kit comprising the same, and uses thereof
WO2018175872A1 (en) * 2017-03-24 2018-09-27 President And Fellows Of Harvard College Methods of genome engineering by nuclease-transposase fusion proteins

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KR20220019794A (ko) 2022-02-17
CN114026240A (zh) 2022-02-08
AU2020290790B2 (en) 2026-02-19
IL288794A (en) 2022-02-01
AU2020290790A1 (en) 2022-01-27
JP2022540318A (ja) 2022-09-15
MX2021015157A (es) 2022-03-17
US20220235379A1 (en) 2022-07-28
CA3141422A1 (en) 2020-12-17
EP3983541A1 (en) 2022-04-20
WO2020250181A1 (en) 2020-12-17
JP7830129B2 (ja) 2026-03-16

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