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
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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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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
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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
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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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    • C12N15/09—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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    • 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
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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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  • 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 (https=)
EP (1) EP3983541A1 (https=)
JP (1) JP7830129B2 (https=)
KR (1) KR20220019794A (https=)
CN (1) CN114026240A (https=)
AU (1) AU2020290790B2 (https=)
BR (1) BR112021024828A2 (https=)
CA (1) CA3141422A1 (https=)
IL (1) IL288794A (https=)
MX (1) MX2021015157A (https=)
WO (1) WO2020250181A1 (https=)

Families Citing this family (15)

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Publication number Priority date Publication date Assignee Title
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
AU2021403660A1 (en) 2020-12-16 2023-07-06 Universitat Pompeu Fabra Programmable transposases and uses thereof
JP2023553701A (ja) * 2020-12-16 2023-12-25 ウニベルシタット ポンペウ ファブラ 先天性筋ジストロフィーの処置のための治療用ｌａｍａ２ペイロード
EP4341392A4 (en) * 2021-05-14 2025-02-19 Becton Dickinson and Company Multiplexed unbiased nucleic acid amplification method
CA3234642A1 (en) * 2021-10-04 2023-04-13 Poseida Therapeutics, Inc. Transposases and uses thereof
JP2024545923A (ja) * 2021-12-30 2024-12-13 リーゲル セラピューティクス，インコーポレイティッド ナトリウム電位依存性チャネルアルファサブユニット１の発現を調節するための組成物、及びその使用
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
JP2026511237A (ja) * 2023-03-27 2026-04-10 ベイジン・アストラジェノミクス・テクノロジー・カンパニー，リミテッド 単離されたトランスポザーゼ及びその使用
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
CN121443733A (zh) 2023-06-01 2026-01-30 英特格拉治疗公司 转座酶及其用途
WO2025212757A2 (en) * 2024-04-03 2025-10-09 Buck Institute For Research On Aging Cas enabled targeted transposition
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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Publication number Priority date Publication date Assignee Title
DE60335637D1 (de) * 2002-07-24 2011-02-17 Manoa Biosciences Inc Vektoren auf transposonbasis und verfahren zur integration von nukleinsäuren
WO2013012824A2 (en) * 2011-07-15 2013-01-24 The Johns Hopkins University Trichoplusia ni piggybac transposases with reduced integration activity
US20170114333A1 (en) * 2014-04-11 2017-04-27 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
JP2018513681A (ja) * 2015-03-31 2018-05-31 エクセリゲン サイエンティフィック, インコーポレイテッドExeligen Scientific, Inc. 細胞または生物のゲノムへのＤＮＡ配列の標的化組み込みのためのＣａｓ９レトロウイルスインテグラーゼおよびＣａｓ９レコンビナーゼ系
KR102249982B1 (ko) * 2015-12-14 2021-05-11 게놈프론티어 테라퓨틱스 인코포레이티드 트랜스포존 시스템, 이를 포함한 키트, 및 이들의 용도
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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MX2021015157A (es) 2022-03-17
JP7830129B2 (ja) 2026-03-16
AU2020290790B2 (en) 2026-02-19
CA3141422A1 (en) 2020-12-17
IL288794A (en) 2022-02-01
KR20220019794A (ko) 2022-02-17
AU2020290790A1 (en) 2022-01-27
WO2020250181A1 (en) 2020-12-17
EP3983541A1 (en) 2022-04-20
CN114026240A (zh) 2022-02-08
JP2022540318A (ja) 2022-09-15
US20220235379A1 (en) 2022-07-28

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