JP2024513967A - 非ウイルス相同性媒介末端結合 - Google Patents

非ウイルス相同性媒介末端結合 Download PDF

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JP2024513967A
JP2024513967A JP2023562658A JP2023562658A JP2024513967A JP 2024513967 A JP2024513967 A JP 2024513967A JP 2023562658 A JP2023562658 A JP 2023562658A JP 2023562658 A JP2023562658 A JP 2023562658A JP 2024513967 A JP2024513967 A JP 2024513967A
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cdl
target sequence
composition
cell
template
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JP2024513967A5 (https=
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アーロン クーパー
ペイ－ケン スー
ロバート ムート
アニー トロン
トーマス ガードナー
ブライアン スー
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アーセナル バイオサイエンシズ インコーポレイテッド
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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]
    • 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
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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
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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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    • C12N15/09—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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    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
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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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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0636—T lymphocytes
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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)
    • C12N9/1276—RNA-directed DNA polymerase (2.7.7.49), i.e. reverse transcriptase or telomerase
    • 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]
    • C12N9/222—Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/93—Ligases (6)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • 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
    • 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
    • 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
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
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    • C12N2510/00—Genetically modified cells
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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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    • C12R2001/00—Microorganisms ; Processes using microorganisms
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    • 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

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  • Enzymes And Modification Thereof (AREA)
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JP2023562658A 2021-04-13 2022-04-13 非ウイルス相同性媒介末端結合 Pending JP2024513967A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163174468P 2021-04-13 2021-04-13
US63/174,468 2021-04-13
PCT/US2022/024703 WO2022221467A1 (en) 2021-04-13 2022-04-13 Non-viral homology mediated end joining

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JP2024513967A true JP2024513967A (ja) 2024-03-27
JP2024513967A5 JP2024513967A5 (https=) 2025-04-21

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US (1) US20240240164A1 (https=)
EP (1) EP4323514A4 (https=)
JP (1) JP2024513967A (https=)
KR (1) KR20230169221A (https=)
CN (1) CN117355607A (https=)
AU (1) AU2022257004A1 (https=)
BR (1) BR112023021283A2 (https=)
CA (1) CA3215080A1 (https=)
CL (2) CL2023003030A1 (https=)
CO (1) CO2023015207A2 (https=)
IL (1) IL307447A (https=)
MX (1) MX2023012044A (https=)
PE (1) PE20231922A1 (https=)
WO (1) WO2022221467A1 (https=)

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IL322949A (en) 2023-03-03 2025-10-01 Arsenal Biosciences Inc Systems targeting PSMA and CA9

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020123871A1 (en) * 2018-12-12 2020-06-18 The Regents Of The University Of California Compositions and methods for modifying a target nucleic acid

Family Cites Families (2)

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Publication number Priority date Publication date Assignee Title
US20170000743A1 (en) * 2015-07-02 2017-01-05 Vindico NanoBio Technology Inc. Compositions and Methods for Delivery of Gene Editing Tools Using Polymeric Vesicles
CN114630910A (zh) * 2019-06-25 2022-06-14 伊纳瑞农业技术有限公司 改善的同源依赖修复基因组编辑

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020123871A1 (en) * 2018-12-12 2020-06-18 The Regents Of The University Of California Compositions and methods for modifying a target nucleic acid

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
GENOME BIOLOGY, vol. 18, no. 35, JPN6026001540, 2017, pages 1 - 18, ISSN: 0005773421 *
HUMAN GENE THERAPY, vol. 30, no. 7, JPN6025040640, 2019, pages 814 - 828, ISSN: 0005773424 *
NATURE BIOTECHNOLOGY, vol. 38, JPN6026001542, 2020, pages 44 - 49, ISSN: 0005773422 *
NATURE, vol. 559, JPN6026001541, 2018, pages 405 - 409, ISSN: 0005773423 *

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BR112023021283A2 (pt) 2023-12-19
CO2023015207A2 (es) 2024-02-05
EP4323514A4 (en) 2025-06-04
US20240240164A1 (en) 2024-07-18
CL2025001317A1 (es) 2025-08-22
AU2022257004A1 (en) 2023-11-23
KR20230169221A (ko) 2023-12-15
EP4323514A1 (en) 2024-02-21
IL307447A (en) 2023-12-01
WO2022221467A1 (en) 2022-10-20
CL2023003030A1 (es) 2024-03-22
PE20231922A1 (es) 2023-11-28
CN117355607A (zh) 2024-01-05
CA3215080A1 (en) 2022-10-20
MX2023012044A (es) 2024-01-05

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