IL273595B2 - Strange cpf1 guide RNA - Google Patents

Strange cpf1 guide RNA

Info

Publication number
IL273595B2
IL273595B2 IL273595A IL27359520A IL273595B2 IL 273595 B2 IL273595 B2 IL 273595B2 IL 273595 A IL273595 A IL 273595A IL 27359520 A IL27359520 A IL 27359520A IL 273595 B2 IL273595 B2 IL 273595B2
Authority
IL
Israel
Prior art keywords
nucleic acid
sequence
crrna
cpf1
extension
Prior art date
Application number
IL273595A
Other languages
English (en)
Hebrew (he)
Other versions
IL273595B1 (en
IL273595A (en
Original Assignee
Genedit Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Genedit Inc filed Critical Genedit Inc
Publication of IL273595A publication Critical patent/IL273595A/en
Publication of IL273595B1 publication Critical patent/IL273595B1/en
Publication of IL273595B2 publication Critical patent/IL273595B2/en

Links

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3519Fusion with another nucleic acid
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin

Landscapes

  • 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)
IL273595A 2017-10-02 2018-10-02 Strange cpf1 guide RNA IL273595B2 (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201762567123P 2017-10-02 2017-10-02
US201862617138P 2018-01-12 2018-01-12
US201862697327P 2018-07-12 2018-07-12
PCT/US2018/054027 WO2019070762A1 (en) 2017-10-02 2018-10-02 RNA GUIDE CPF1 MODIFIED

Publications (3)

Publication Number Publication Date
IL273595A IL273595A (en) 2020-05-31
IL273595B1 IL273595B1 (en) 2025-10-01
IL273595B2 true IL273595B2 (en) 2026-02-01

Family

ID=64184173

Family Applications (2)

Application Number Title Priority Date Filing Date
IL273595A IL273595B2 (en) 2017-10-02 2018-10-02 Strange cpf1 guide RNA
IL323269A IL323269A (en) 2017-10-02 2025-09-10 Strange cpf1 guide RNA

Family Applications After (1)

Application Number Title Priority Date Filing Date
IL323269A IL323269A (en) 2017-10-02 2025-09-10 Strange cpf1 guide RNA

Country Status (12)

Country Link
US (1) US12606823B2 (https=)
EP (1) EP3692154A1 (https=)
JP (2) JP2020537540A (https=)
KR (2) KR102907044B1 (https=)
CN (2) CN120290559A (https=)
AU (2) AU2018345683B2 (https=)
BR (1) BR112020006601A2 (https=)
CA (1) CA3077189A1 (https=)
IL (2) IL273595B2 (https=)
MX (1) MX2020003339A (https=)
SG (1) SG11202003083PA (https=)
WO (1) WO2019070762A1 (https=)

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GB201616590D0 (en) 2016-09-29 2016-11-16 Oxford Nanopore Technologies Limited Method
EP3596217A1 (en) 2017-03-14 2020-01-22 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
EP3622070A2 (en) 2017-05-10 2020-03-18 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
CN120290559A (zh) 2017-10-02 2025-07-11 基因编辑有限公司 修饰的cpf1引导rna
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
EP4076479A4 (en) * 2019-12-18 2024-05-22 Editas Medicine, Inc. GENETICALLY MODIFIED 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
CN116848240A (zh) * 2020-12-07 2023-10-03 因思科瑞普特公司 核酸引导的切口酶编辑中的gRNA稳定
IL316048A (en) * 2022-04-13 2024-11-01 Caribou Biosciences Inc Therapeutic applications of V-type CRISPR systems
US20240167029A1 (en) * 2022-06-27 2024-05-23 University Of Massachusetts Modified guide rnas for crispr genome editing
IL324634A (en) 2023-05-15 2026-01-01 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

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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
WO2017070598A1 (en) * 2015-10-23 2017-04-27 Caribou Biosciences, Inc. Engineered crispr class 2 cross-type nucleic-acid targeting nucleic acids

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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
EP3241902B1 (en) 2012-05-25 2018-02-28 The Regents of The University of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
US20140349400A1 (en) 2013-03-15 2014-11-27 Massachusetts Institute Of Technology Programmable Modification of DNA
US10563225B2 (en) 2013-07-26 2020-02-18 President And Fellows Of Harvard College Genome engineering
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
WO2016022866A1 (en) 2014-08-07 2016-02-11 Agilent Technologies, Inc. Cis-blocked guide rna
EP3183358B1 (en) 2014-08-19 2020-10-07 President and Fellows of Harvard College Rna-guided systems for probing and mapping of nucleic acids
US20170306306A1 (en) 2014-10-24 2017-10-26 Life Technologies Corporation Compositions and Methods for Enhancing Homologous Recombination
US10900034B2 (en) 2014-12-03 2021-01-26 Agilent Technologies, Inc. Guide RNA with chemical modifications
EP3889260A1 (en) 2014-12-12 2021-10-06 The Broad Institute, Inc. Protected guide rnas (pgrnas)
AU2016261358B2 (en) 2015-05-11 2021-09-16 Editas Medicine, Inc. Optimized CRISPR/Cas9 systems and methods for gene editing in stem cells
WO2016183402A2 (en) 2015-05-13 2016-11-17 President And Fellows Of Harvard College Methods of making and using guide rna for use with cas9 systems
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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
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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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Also Published As

Publication number Publication date
EP3692154A1 (en) 2020-08-12
AU2018345683A1 (en) 2020-04-23
US12606823B2 (en) 2026-04-21
US20200299689A1 (en) 2020-09-24
CN111770994B (zh) 2025-03-18
IL273595B1 (en) 2025-10-01
AU2018345683B2 (en) 2025-05-08
MX2020003339A (es) 2020-11-06
CA3077189A1 (en) 2019-04-11
CN120290559A (zh) 2025-07-11
SG11202003083PA (en) 2020-05-28
JP2020537540A (ja) 2020-12-24
WO2019070762A1 (en) 2019-04-11
IL273595A (en) 2020-05-31
BR112020006601A2 (pt) 2020-12-08
KR102907044B1 (ko) 2026-01-02
KR20260011199A (ko) 2026-01-22
AU2025202594A1 (en) 2025-05-22
IL323269A (en) 2025-11-01
KR20200106485A (ko) 2020-09-14
CN111770994A (zh) 2020-10-13
AU2025202594A9 (en) 2025-09-04
JP2024116119A (ja) 2024-08-27

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