CN117795065A - 使用细胞穿透crispr-cas系统在原代免疫细胞中进行基因编辑 - Google Patents

使用细胞穿透crispr-cas系统在原代免疫细胞中进行基因编辑 Download PDF

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Publication number
CN117795065A
CN117795065A CN202280053796.XA CN202280053796A CN117795065A CN 117795065 A CN117795065 A CN 117795065A CN 202280053796 A CN202280053796 A CN 202280053796A CN 117795065 A CN117795065 A CN 117795065A
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CN202280053796.XA
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English (en)
Chinese (zh)
Inventor
S·L·伯格
E·J·惠里
史俊炜
陈则宇
张珍
R·M·科利
J·B·帕克
A·E·巴克斯特
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University of Pennsylvania Penn
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University of Pennsylvania Penn
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Publication of CN117795065A publication Critical patent/CN117795065A/zh
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/06—Fusion polypeptide containing a localisation/targetting motif containing a lysosomal/endosomal localisation signal
    • 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
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/10—Fusion polypeptide containing a localisation/targetting motif containing a tag for extracellular membrane crossing, e.g. TAT or VP22
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CN202280053796.XA 2021-06-02 2022-06-02 使用细胞穿透crispr-cas系统在原代免疫细胞中进行基因编辑 Pending CN117795065A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163196144P 2021-06-02 2021-06-02
US63/196,144 2021-06-02
PCT/US2022/031983 WO2022256546A2 (en) 2021-06-02 2022-06-02 Gene editing in primary immune cells using cell penetrating crispr-cas system

Publications (1)

Publication Number Publication Date
CN117795065A true CN117795065A (zh) 2024-03-29

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CN202280053796.XA Pending CN117795065A (zh) 2021-06-02 2022-06-02 使用细胞穿透crispr-cas系统在原代免疫细胞中进行基因编辑

Country Status (7)

Country Link
US (1) US20240263159A1 (https=)
EP (1) EP4347811A4 (https=)
JP (1) JP2024520644A (https=)
CN (1) CN117795065A (https=)
AU (1) AU2022283895A1 (https=)
CA (1) CA3221125A1 (https=)
WO (1) WO2022256546A2 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN117285599B (zh) * 2023-10-11 2024-04-09 东北农业大学 一种抗胞内细菌的细胞穿透型抗菌肽5vt及其制备方法和应用
WO2025208177A1 (en) * 2024-04-03 2025-10-09 Macquarie University Therapeutic peptides

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10258697B2 (en) * 2014-10-29 2019-04-16 Massachusetts Eye And Ear Infirmary Efficient delivery of therapeutic molecules in vitro and in vivo
WO2017175072A1 (en) * 2016-04-08 2017-10-12 Feldan Bio Inc. Peptide shuttle based gene disruption
US12454685B2 (en) * 2019-07-08 2025-10-28 The Regents Of The University Of California Variant type V CRISPR/Cas effector polypeptides and methods of use thereof

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Publication number Publication date
CA3221125A1 (en) 2022-12-08
JP2024520644A (ja) 2024-05-24
WO2022256546A3 (en) 2023-01-12
EP4347811A4 (en) 2025-06-11
WO2022256546A2 (en) 2022-12-08
AU2022283895A1 (en) 2023-12-14
EP4347811A2 (en) 2024-04-10
AU2022283895A9 (en) 2024-01-25
US20240263159A1 (en) 2024-08-08

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