JP2024513087A - 部位特異的改変のための組成物及び方法 - Google Patents

部位特異的改変のための組成物及び方法 Download PDF

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JP2024513087A
JP2024513087A JP2023561238A JP2023561238A JP2024513087A JP 2024513087 A JP2024513087 A JP 2024513087A JP 2023561238 A JP2023561238 A JP 2023561238A JP 2023561238 A JP2023561238 A JP 2023561238A JP 2024513087 A JP2024513087 A JP 2024513087A
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dna
sequence
polynucleotide
cas
composition
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JP2024513087A5 (https=
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リィ，ソンユエン
マレスカ，マルチェッロ
スヴィコヴィッチ，サーシャ
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AstraZeneca AB
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AstraZeneca AB
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    • C—CHEMISTRY; METALLURGY
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
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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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    • 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
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/93—Ligases (6)
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/07—Nucleotidyltransferases (2.7.7)
    • C12Y207/07049—RNA-directed DNA polymerase (2.7.7.49), i.e. telomerase or reverse-transcriptase
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y605/00—Ligases forming phosphoric ester bonds (6.5)
    • C12Y605/01—Ligases forming phosphoric ester bonds (6.5) forming phosphoric ester bonds (6.5.1)
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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]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/332—Abasic residue
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid

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  • Health & Medical Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • Medicinal Chemistry (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
JP2023561238A 2021-04-07 2022-04-06 部位特異的改変のための組成物及び方法 Pending JP2024513087A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163171651P 2021-04-07 2021-04-07
US63/171,651 2021-04-07
US202163292144P 2021-12-21 2021-12-21
US63/292,144 2021-12-21
PCT/EP2022/059070 WO2022214522A2 (en) 2021-04-07 2022-04-06 Compositions and methods for site-specific modification

Publications (2)

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JP2024513087A true JP2024513087A (ja) 2024-03-21
JP2024513087A5 JP2024513087A5 (https=) 2025-04-15

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US (1) US20240182890A1 (https=)
EP (1) EP4320234A2 (https=)
JP (1) JP2024513087A (https=)
WO (1) WO2022214522A2 (https=)

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* Cited by examiner, † Cited by third party
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EP4133069A2 (en) * 2020-04-08 2023-02-15 Astrazeneca AB Compositions and methods for improved site-specific modification
CN121002177A (zh) * 2023-03-10 2025-11-21 先正达农作物保护股份公司 用于位点特异性整合的cas融合蛋白和相关方法
WO2025035110A1 (en) * 2023-08-10 2025-02-13 Linearx, Inc. Compositions and methods for in vitro transcription
WO2026083309A1 (en) 2024-10-17 2026-04-23 Astrazeneca Ab Use of inhibitors or modulators of artemis in gene editing

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WO2018094356A2 (en) * 2016-11-18 2018-05-24 Genedit Inc. Compositions and methods for target nucleic acid modification
WO2018138385A1 (en) * 2017-01-30 2018-08-02 Kws Saat Se Repair template linkage to endonucleases for genome engineering
WO2019051097A1 (en) * 2017-09-08 2019-03-14 The Regents Of The University Of California RNA-GUIDED ENDONUCLEASE FUSION POLYPEPTIDES AND METHODS OF USING SAME
WO2019204226A1 (en) * 2018-04-16 2019-10-24 University Of Massachusetts Compositions and methods for improved gene editing
WO2020191248A1 (en) * 2019-03-19 2020-09-24 The Broad Institute, Inc. Method and compositions for editing nucleotide sequences
JP2021503279A (ja) * 2017-11-16 2021-02-12 アストラゼネカ・アクチエボラーグＡｓｔｒａｚｅｎｅｃａ Ａｋｔｉｅｂｏｌａｇ Ｃａｓ９ベースノックイン方針の効力を改善するための組成物及び方法
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WO2022214522A2 (en) 2022-10-13
WO2022214522A3 (en) 2022-11-17
EP4320234A2 (en) 2024-02-14

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