KR20230122628A - 부위-지향 돌연변이 유발을 위한 조성물 및 방법 - Google Patents

부위-지향 돌연변이 유발을 위한 조성물 및 방법 Download PDF

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Publication number
KR20230122628A
KR20230122628A KR1020237024378A KR20237024378A KR20230122628A KR 20230122628 A KR20230122628 A KR 20230122628A KR 1020237024378 A KR1020237024378 A KR 1020237024378A KR 20237024378 A KR20237024378 A KR 20237024378A KR 20230122628 A KR20230122628 A KR 20230122628A
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South Korea
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cell
target site
deletions
dna
fusion polypeptide
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KR1020237024378A
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Korean (ko)
Inventor
조던 자르주르
앤-레이첼 크로스택
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2세븐티 바이오, 인코포레이티드
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Publication of KR20230122628A publication Critical patent/KR20230122628A/ko
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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/14Blood; Artificial blood
    • A61K35/17Lymphocytes; B-cells; T-cells; Natural killer cells; Interferon-activated or cytokine-activated lymphocytes
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/465Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
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    • C12N5/0602Vertebrate cells
    • C12N5/0634Cells from the blood or the immune system
    • C12N5/0636T lymphocytes
    • C12N5/0638Cytotoxic T lymphocytes [CTL] or lymphokine activated killer cells [LAK]
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    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
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    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/11Exodeoxyribonucleases producing 5'-phosphomonoesters (3.1.11)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K38/00Medicinal preparations containing peptides
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    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
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    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/80Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
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    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/80Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
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    • C12N2510/00Genetically modified cells
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    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/11Exodeoxyribonucleases producing 5'-phosphomonoesters (3.1.11)
    • C12Y301/11001Exodeoxyribonuclease I (3.1.11.1)
    • CCHEMISTRY; METALLURGY
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    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/11Exodeoxyribonucleases producing 5'-phosphomonoesters (3.1.11)
    • C12Y301/11002Exodeoxyribonuclease III (3.1.11.2)

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  • Bioinformatics & Cheminformatics (AREA)
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  • Wood Science & Technology (AREA)
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  • General Engineering & Computer Science (AREA)
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  • Biochemistry (AREA)
  • Cell Biology (AREA)
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  • Developmental Biology & Embryology (AREA)
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  • Physics & Mathematics (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Agricultural Chemicals And Associated Chemicals (AREA)
KR1020237024378A 2020-12-21 2021-12-16 부위-지향 돌연변이 유발을 위한 조성물 및 방법 Pending KR20230122628A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063128391P 2020-12-21 2020-12-21
US63/128,391 2020-12-21
PCT/US2021/063771 WO2022140150A1 (en) 2020-12-21 2021-12-16 Compositions and methods for site-directed mutagenesis

Publications (1)

Publication Number Publication Date
KR20230122628A true KR20230122628A (ko) 2023-08-22

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Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020237024378A Pending KR20230122628A (ko) 2020-12-21 2021-12-16 부위-지향 돌연변이 유발을 위한 조성물 및 방법

Country Status (8)

Country Link
US (1) US20240336904A1 (https=)
EP (1) EP4262850A4 (https=)
JP (1) JP2024503249A (https=)
KR (1) KR20230122628A (https=)
CN (1) CN116887853A (https=)
AU (1) AU2021410632A1 (https=)
CA (1) CA3202889A1 (https=)
WO (1) WO2022140150A1 (https=)

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012118717A2 (en) * 2011-02-28 2012-09-07 Seattle Children's Research Institute Coupling endonucleases with end-processing enzymes drive high efficiency gene disruption
RU2650811C2 (ru) * 2012-02-24 2018-04-17 Фред Хатчинсон Кэнсер Рисерч Сентер Композиции и способы лечения гемоглобинопатии
US10000746B2 (en) * 2013-05-31 2018-06-19 Cellectis LAGLIDADG homing endonuclease cleaving the T cell receptor alpha gene and uses thereof
US20180119174A1 (en) * 2015-05-13 2018-05-03 Seattle Children's Hospita (dba Seattle Children's Research Institute Enhancing endonuclease based gene editing in primary cells
MX2018010924A (es) * 2016-03-11 2019-02-13 Bluebird Bio Inc Células efectoras inmunitarias con edición genómica.
WO2018071565A1 (en) * 2016-10-11 2018-04-19 Bluebird Bio, Inc. TCRa HOMING ENDONUCLEASE VARIANTS
IL313037A (en) * 2017-05-25 2024-07-01 2Seventy Bio Inc Cblb endonuclease variants, compositions, and methods of use
CN112533627A (zh) * 2018-03-27 2021-03-19 G+Flas 生命科学公司 序列特异性体内细胞靶向
WO2020072059A1 (en) * 2018-10-04 2020-04-09 Bluebird Bio, Inc. Cblb endonuclease variants, compositions, and methods of use
WO2020123371A2 (en) * 2018-12-10 2020-06-18 Bluebird Bio, Inc. Homing endonuclease variants
US20220280567A1 (en) * 2019-06-14 2022-09-08 2Seventy Bio, Inc. Compositions and methods for treating cancer

Also Published As

Publication number Publication date
JP2024503249A (ja) 2024-01-25
WO2022140150A1 (en) 2022-06-30
EP4262850A4 (en) 2025-07-09
EP4262850A1 (en) 2023-10-25
AU2021410632A1 (en) 2023-08-03
CA3202889A1 (en) 2022-06-30
CN116887853A (zh) 2023-10-13
US20240336904A1 (en) 2024-10-10

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