CN111770994B - 修饰的cpf1引导rna - Google Patents

修饰的cpf1引导rna Download PDF

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Publication number
CN111770994B
CN111770994B CN201880077637.7A CN201880077637A CN111770994B CN 111770994 B CN111770994 B CN 111770994B CN 201880077637 A CN201880077637 A CN 201880077637A CN 111770994 B CN111770994 B CN 111770994B
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sequence
nucleic acid
crrna
cpf1
extension
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CN111770994A (zh
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K·李
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Gene Editing Ltd
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Gene Editing Ltd
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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
    • 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
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • 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
    • C12N15/87—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
    • 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]
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin

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  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
CN201880077637.7A 2017-10-02 2018-10-02 修饰的cpf1引导rna Active CN111770994B (zh)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CN202510240123.0A CN120290559A (zh) 2017-10-02 2018-10-02 修饰的cpf1引导rna

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201762567123P 2017-10-02 2017-10-02
US62/567,123 2017-10-02
US201862617138P 2018-01-12 2018-01-12
US62/617,138 2018-01-12
US201862697327P 2018-07-12 2018-07-12
US62/697,327 2018-07-12
PCT/US2018/054027 WO2019070762A1 (en) 2017-10-02 2018-10-02 RNA GUIDE CPF1 MODIFIED

Related Child Applications (1)

Application Number Title Priority Date Filing Date
CN202510240123.0A Division CN120290559A (zh) 2017-10-02 2018-10-02 修饰的cpf1引导rna

Publications (2)

Publication Number Publication Date
CN111770994A CN111770994A (zh) 2020-10-13
CN111770994B true CN111770994B (zh) 2025-03-18

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CN201880077637.7A Active CN111770994B (zh) 2017-10-02 2018-10-02 修饰的cpf1引导rna
CN202510240123.0A Pending CN120290559A (zh) 2017-10-02 2018-10-02 修饰的cpf1引导rna

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US (1) US20200299689A1 (enExample)
EP (1) EP3692154A1 (enExample)
JP (2) JP2020537540A (enExample)
CN (2) CN111770994B (enExample)
AU (2) AU2018345683B2 (enExample)
BR (1) BR112020006601A2 (enExample)
CA (1) CA3077189A1 (enExample)
IL (2) IL273595B1 (enExample)
MX (1) MX2020003339A (enExample)
SG (1) SG11202003083PA (enExample)
WO (1) WO2019070762A1 (enExample)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018170184A1 (en) 2017-03-14 2018-09-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
WO2018209158A2 (en) 2017-05-10 2018-11-15 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
US20220177957A1 (en) * 2020-12-07 2022-06-09 Inscripta, Inc. gRNA STABILIZATION IN NUCLEIC ACID-GUIDED NICKASE EDITING
EP3652312A1 (en) 2017-07-14 2020-05-20 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
US11268092B2 (en) 2018-01-12 2022-03-08 GenEdit, Inc. Structure-engineered guide RNA
WO2019178426A1 (en) * 2018-03-14 2019-09-19 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
EP3823633A4 (en) 2018-06-29 2023-05-03 Editas Medicine, Inc. SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO
GB201905651D0 (en) * 2019-04-24 2019-06-05 Lightbio Ltd Nucleic acid constructs and methods for their manufacture
GB201913997D0 (en) * 2019-09-27 2019-11-13 Oxford Nanopore Tech Ltd Method
US20230053028A1 (en) * 2019-12-18 2023-02-16 Editas Medicine, Inc. Engineered cells for therapy
CN113234701B (zh) * 2020-10-20 2022-08-16 珠海舒桐医疗科技有限公司 一种Cpf1蛋白及基因编辑系统
WO2023201270A2 (en) * 2022-04-13 2023-10-19 Caribou Biosciences, Inc. Therapeutic applications of crispr type v systems
US20240167029A1 (en) * 2022-06-27 2024-05-23 University Of Massachusetts Modified guide rnas for crispr genome editing
AU2024270764A1 (en) 2023-05-15 2025-12-04 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
WO2025212120A1 (en) * 2024-04-05 2025-10-09 Arbor Biotechnologies, Inc. Chemical modifications of guide rnas for crispr nucleases

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN106244591A (zh) * 2016-08-23 2016-12-21 苏州吉玛基因股份有限公司 修饰crRNA在CRISPR/Cpf1基因编辑系统中的应用

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US4863457A (en) 1986-11-24 1989-09-05 Lee David A Drug delivery device
US5378475A (en) 1991-02-21 1995-01-03 University Of Kentucky Research Foundation Sustained release drug delivery devices
US5443505A (en) 1993-11-15 1995-08-22 Oculex Pharmaceuticals, Inc. Biocompatible ocular implants
ES2699848T3 (es) * 2015-10-23 2019-02-13 Caribou Biosciences Inc Acido nucleico CRISPR clase 2 de tipo cruzado modificado que se dirige a ácidos nucleicos
CA3062165A1 (en) * 2017-05-04 2018-11-08 The Trustees Of The University Of Pennsylvania Compositions and methods for gene editing in t cells using crispr/cpf1

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN106244591A (zh) * 2016-08-23 2016-12-21 苏州吉玛基因股份有限公司 修饰crRNA在CRISPR/Cpf1基因编辑系统中的应用

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Engineering CRISPR-Cpf1 crRNAs and mRNAs to maximize genome editing efficiency;Bin等;《Nat Biomed Eng》;图1-图3 *
Generation of targeted mutant rice using a CRISPR-Cpf1 system;Rongfang等;《Plant Biotechnology Journal》;图1 *

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EP3692154A1 (en) 2020-08-12
CA3077189A1 (en) 2019-04-11
US20200299689A1 (en) 2020-09-24
SG11202003083PA (en) 2020-05-28
WO2019070762A1 (en) 2019-04-11
MX2020003339A (es) 2020-11-06
JP2024116119A (ja) 2024-08-27
CN120290559A (zh) 2025-07-11
AU2018345683A1 (en) 2020-04-23
IL273595B1 (en) 2025-10-01
KR20200106485A (ko) 2020-09-14
IL273595A (en) 2020-05-31
IL323269A (en) 2025-11-01
JP2020537540A (ja) 2020-12-24
CN111770994A (zh) 2020-10-13
BR112020006601A2 (pt) 2020-12-08
AU2025202594A9 (en) 2025-09-04
AU2025202594A1 (en) 2025-05-22
AU2018345683B2 (en) 2025-05-08

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