JP2020537540A - 改変ｃｐｆ１ガイドｒｎａ - Google Patents

改変ｃｐｆ１ガイドｒｎａ Download PDF

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Publication number
JP2020537540A
JP2020537540A JP2020539688A JP2020539688A JP2020537540A JP 2020537540 A JP2020537540 A JP 2020537540A JP 2020539688 A JP2020539688 A JP 2020539688A JP 2020539688 A JP2020539688 A JP 2020539688A JP 2020537540 A JP2020537540 A JP 2020537540A
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Japan
Prior art keywords
nucleic acid
sequence
crrna
cpf1
extension
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JP2020539688A
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Japanese (ja)
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JP2020537540A5 (cg-RX-API-DMAC7.html
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リー、クンウー
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ジーンエディット インコーポレイテッド
ジーンエディット インコーポレイテッド
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Publication of JP2020537540A publication Critical patent/JP2020537540A/ja
Publication of JP2020537540A5 publication Critical patent/JP2020537540A5/ja
Priority to JP2024076160A priority Critical patent/JP2024116119A/ja
Pending legal-status Critical Current

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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)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
JP2020539688A 2017-10-02 2018-10-02 改変ｃｐｆ１ガイドｒｎａ Pending JP2020537540A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024076160A JP2024116119A (ja) 2017-10-02 2024-05-08 改変ｃｐｆ１ガイド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
JP2024076160A Division JP2024116119A (ja) 2017-10-02 2024-05-08 改変ｃｐｆ１ガイドｒｎａ

Publications (2)

Publication Number Publication Date
JP2020537540A true JP2020537540A (ja) 2020-12-24
JP2020537540A5 JP2020537540A5 (cg-RX-API-DMAC7.html) 2021-11-11

Family

ID=64184173

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2020539688A Pending JP2020537540A (ja) 2017-10-02 2018-10-02 改変ｃｐｆ１ガイドｒｎａ
JP2024076160A Pending JP2024116119A (ja) 2017-10-02 2024-05-08 改変ｃｐｆ１ガイドｒｎａ

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024076160A Pending JP2024116119A (ja) 2017-10-02 2024-05-08 改変ｃｐｆ１ガイドｒｎａ

Country Status (11)

Country Link
US (1) US20200299689A1 (cg-RX-API-DMAC7.html)
EP (1) EP3692154A1 (cg-RX-API-DMAC7.html)
JP (2) JP2020537540A (cg-RX-API-DMAC7.html)
CN (2) CN111770994B (cg-RX-API-DMAC7.html)
AU (2) AU2018345683B2 (cg-RX-API-DMAC7.html)
BR (1) BR112020006601A2 (cg-RX-API-DMAC7.html)
CA (1) CA3077189A1 (cg-RX-API-DMAC7.html)
IL (2) IL273595B1 (cg-RX-API-DMAC7.html)
MX (1) MX2020003339A (cg-RX-API-DMAC7.html)
SG (1) SG11202003083PA (cg-RX-API-DMAC7.html)
WO (1) WO2019070762A1 (cg-RX-API-DMAC7.html)

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
US11866726B2 (en) 2017-07-14 2024-01-09 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
CA3104856A1 (en) 2018-06-29 2020-01-02 Editas Medicine, Inc. Synthetic guide molecules, compositions and methods relating 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
WO2021127594A1 (en) * 2019-12-18 2021-06-24 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
WO2022125329A1 (en) * 2020-12-07 2022-06-16 Inscripta, Inc. gRNA STABILIZATION IN NUCLEIC ACID-GUIDED NICKASE EDITING
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

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
DK3350327T3 (en) * 2015-10-23 2019-01-21 Caribou Biosciences Inc CONSTRUCTED CRISPR CLASS-2-NUCLEIC ACID TARGETING-NUCLEIC ACID
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 (3)

* Cited by examiner, † Cited by third party
Title
ELIFE 2017, MAY 2, VOL. 6, ARTICLE NO. E25312 (PP. 1-17), JPN6022042897, ISSN: 0005231708 *
GENOME BIOL, 2015 DEC 15, VOL. 16, ARTICLE NO. 280 (PP. 1-10), JPN6022042898, ISSN: 0005231709 *
NATURE, 2016 APR 20, VOL. 532, NO. 7600, PP. 517-521, JPN6022042895, ISSN: 0005052260 *

Also Published As

Publication number Publication date
CA3077189A1 (en) 2019-04-11
IL323269A (en) 2025-11-01
AU2025202594A1 (en) 2025-05-22
CN120290559A (zh) 2025-07-11
JP2024116119A (ja) 2024-08-27
WO2019070762A1 (en) 2019-04-11
SG11202003083PA (en) 2020-05-28
AU2018345683B2 (en) 2025-05-08
AU2025202594A9 (en) 2025-09-04
IL273595A (en) 2020-05-31
MX2020003339A (es) 2020-11-06
IL273595B1 (en) 2025-10-01
CN111770994B (zh) 2025-03-18
KR20200106485A (ko) 2020-09-14
EP3692154A1 (en) 2020-08-12
BR112020006601A2 (pt) 2020-12-08
US20200299689A1 (en) 2020-09-24
CN111770994A (zh) 2020-10-13
AU2018345683A1 (en) 2020-04-23

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