JP2020537540A - 改変ｃｐｆ１ガイドｒｎａ - Google Patents

改変ｃｐｆ１ガイドｒｎａ Download PDF

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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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nucleic acid
sequence
crrna
cpf1
extension
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JP2020539688A
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Japanese (ja)
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JP2020537540A5 (enExample
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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
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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
    • 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
    • C12N9/226—Class 2 CAS enzyme complex, e.g. single CAS protein
    • 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)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (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 (enExample) 2021-11-11

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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

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JP2024076160A Pending JP2024116119A (ja) 2017-10-02 2024-05-08 改変ｃｐｆ１ガイドｒｎａ

Country Status (12)

Country Link
US (1) US12606823B2 (enExample)
EP (1) EP3692154A1 (enExample)
JP (2) JP2020537540A (enExample)
KR (2) KR102907044B1 (enExample)
CN (2) CN111770994B (enExample)
AU (2) AU2018345683B2 (enExample)
BR (1) BR112020006601A2 (enExample)
CA (1) CA3077189A1 (enExample)
IL (2) IL273595B2 (enExample)
MX (1) MX2020003339A (enExample)
SG (1) SG11202003083PA (enExample)
WO (1) WO2019070762A1 (enExample)

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EP3622070A2 (en) 2017-05-10 2020-03-18 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
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
KR102907044B1 (ko) 2017-10-02 2026-01-02 진에딧 인코포레이티드 변형된 cpf1 가이드 rna
US11268092B2 (en) 2018-01-12 2022-03-08 GenEdit, Inc. Structure-engineered guide RNA
IL277234B2 (en) * 2018-03-14 2026-03-01 Editas Medicine Inc Systems and methods for treating hemoglobin diseases
AU2019291918B2 (en) 2018-06-29 2025-06-12 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
CN114929250A (zh) * 2019-12-18 2022-08-19 爱迪塔斯医药公司 用于疗法的经工程化的细胞
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
US20250325698A1 (en) * 2022-04-13 2025-10-23 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
JP2026523341A (ja) 2023-05-15 2026-07-14 エヌクロマ・バイオ，インコーポレーテッド Ｈｂｖ遺伝子発現のエピジェネティック調節のための組成物および方法
WO2025212120A1 (en) * 2024-04-05 2025-10-09 Arbor Biotechnologies, Inc. Chemical modifications of guide rnas for crispr nucleases

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Also Published As

Publication number Publication date
KR102907044B1 (ko) 2026-01-02
AU2025202594A1 (en) 2025-05-22
CN111770994A (zh) 2020-10-13
AU2018345683B2 (en) 2025-05-08
US12606823B2 (en) 2026-04-21
SG11202003083PA (en) 2020-05-28
BR112020006601A2 (pt) 2020-12-08
AU2018345683A1 (en) 2020-04-23
IL323269A (en) 2025-11-01
US20200299689A1 (en) 2020-09-24
CN120290559A (zh) 2025-07-11
AU2025202594A9 (en) 2025-09-04
IL273595B2 (en) 2026-02-01
IL273595A (en) 2020-05-31
KR20260011199A (ko) 2026-01-22
IL273595B1 (en) 2025-10-01
EP3692154A1 (en) 2020-08-12
CN111770994B (zh) 2025-03-18
JP2024116119A (ja) 2024-08-27
KR20200106485A (ko) 2020-09-14
MX2020003339A (es) 2020-11-06
CA3077189A1 (en) 2019-04-11
WO2019070762A1 (en) 2019-04-11

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