SG11202103722TA - Programmable dna base editing by nme2cas9-deaminase fusion proteins - Google Patents

Programmable dna base editing by nme2cas9-deaminase fusion proteins

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Publication number
SG11202103722TA
SG11202103722TA SG11202103722TA SG11202103722TA SG11202103722TA SG 11202103722T A SG11202103722T A SG 11202103722TA SG 11202103722T A SG11202103722T A SG 11202103722TA SG 11202103722T A SG11202103722T A SG 11202103722TA SG 11202103722T A SG11202103722T A SG 11202103722TA
Authority
SG
Singapore
Prior art keywords
nme2cas9
fusion proteins
dna base
base editing
programmable dna
Prior art date
Application number
SG11202103722TA
Other languages
English (en)
Inventor
Erik Sontheimer
Xin Gao
Aamir Mir
Alireza Edraki
Scot Wolfe
Pengpeng Liu
Original Assignee
Univ Massachusetts
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 Univ Massachusetts filed Critical Univ Massachusetts
Publication of SG11202103722TA publication Critical patent/SG11202103722TA/en

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    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases RNAses, DNAses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • CCHEMISTRY; METALLURGY
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    • 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
    • C12N15/62DNA sequences coding for fusion proteins
    • C12N15/625DNA sequences coding for fusion proteins containing a sequence coding for a signal sequence
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • CCHEMISTRY; METALLURGY
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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/78Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y305/00Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04004Adenosine deaminase (3.5.4.4)
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    • C12YENZYMES
    • C12Y305/00Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04005Cytidine deaminase (3.5.4.5)
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • CCHEMISTRY; METALLURGY
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    • 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 [CRISPRs]
    • CCHEMISTRY; METALLURGY
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14142Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • 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
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171Demonstrated in vivo effect
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2800/00Nucleic acids vectors
    • C12N2800/80Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
SG11202103722TA 2018-10-15 2019-10-15 Programmable dna base editing by nme2cas9-deaminase fusion proteins SG11202103722TA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862745666P 2018-10-15 2018-10-15
PCT/US2019/056341 WO2020081568A1 (en) 2018-10-15 2019-10-15 Programmable dna base editing by nme2cas9-deaminase fusion proteins

Publications (1)

Publication Number Publication Date
SG11202103722TA true SG11202103722TA (en) 2021-05-28

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202103722TA SG11202103722TA (en) 2018-10-15 2019-10-15 Programmable dna base editing by nme2cas9-deaminase fusion proteins

Country Status (14)

Country Link
US (1) US20220290113A1 (pt)
EP (1) EP3867367A1 (pt)
JP (1) JP2022508716A (pt)
KR (1) KR20210077732A (pt)
CN (1) CN113166743A (pt)
AU (1) AU2019362874A1 (pt)
BR (1) BR112021007123A2 (pt)
CA (1) CA3116555A1 (pt)
CO (1) CO2021006336A2 (pt)
EA (1) EA202191033A1 (pt)
IL (1) IL282267A (pt)
MX (1) MX2021004301A (pt)
SG (1) SG11202103722TA (pt)
WO (1) WO2020081568A1 (pt)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2021267940A1 (en) 2020-05-08 2022-12-08 President And Fellows Of Harvard College Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
GB202010348D0 (en) 2020-07-06 2020-08-19 Univ Wageningen Base editing tools
WO2022125968A1 (en) 2020-12-11 2022-06-16 Intellia Therapeutics, Inc. Polynucleotides, compositions, and methods for genome editing involving deamination
AU2021396403A1 (en) 2020-12-11 2023-06-29 Intellia Therapeutics, Inc. Compositions and methods for reducing mhc class ii in a cell
WO2022140587A1 (en) 2020-12-23 2022-06-30 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying ciita in a cell
CR20230320A (es) 2020-12-23 2023-10-23 Intellia Therapeutics Inc Composiciones y métodos para reducir los hla-a en una célula
WO2023081070A1 (en) * 2021-11-02 2023-05-11 University Of Massachusetts Nme2Cas9 INLAID DOMAIN FUSION PROTEINS
AU2022382975A1 (en) 2021-11-03 2024-05-02 Intellia Therapeutics, Inc. Polynucleotides, compositions, and methods for genome editing
WO2023196802A1 (en) * 2022-04-04 2023-10-12 The Broad Institute, Inc. Cas9 variants having non-canonical pam specificities and uses thereof
WO2023227669A2 (en) * 2022-05-26 2023-11-30 UCB Biopharma SRL Novel nucleic acid-editing proteins
WO2023245113A1 (en) 2022-06-16 2023-12-21 Intellia Therapeutics, Inc. Methods and compositions for genetically modifying a cell
WO2023245109A2 (en) 2022-06-16 2023-12-21 Intellia Therapeutics, Inc. Compositions and methods for genomic editing
WO2023245108A2 (en) 2022-06-16 2023-12-21 Intellia Therapeutics, Inc. Compositions and methods for reducing mhc class i in a cell
CN115206456B (zh) * 2022-07-13 2023-04-25 黑龙江大学 基于属性编辑流的分子生成方法
CN115851775B (zh) * 2022-10-18 2023-08-04 哈尔滨工业大学 一种Cas9蛋白抑制剂及其应用
CN115716880A (zh) * 2022-12-07 2023-02-28 云舟生物科技(广州)股份有限公司 一种核定位荧光蛋白及其应用

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NL154598B (nl) 1970-11-10 1977-09-15 Organon Nv Werkwijze voor het aantonen en bepalen van laagmoleculire verbindingen en van eiwitten die deze verbindingen specifiek kunnen binden, alsmede testverpakking.
US3817837A (en) 1971-05-14 1974-06-18 Syva Corp Enzyme amplification assay
US3939350A (en) 1974-04-29 1976-02-17 Board Of Trustees Of The Leland Stanford Junior University Fluorescent immunoassay employing total reflection for activation
US3996345A (en) 1974-08-12 1976-12-07 Syva Company Fluorescence quenching with immunological pairs in immunoassays
US4277437A (en) 1978-04-05 1981-07-07 Syva Company Kit for carrying out chemically induced fluorescence immunoassay
US4275149A (en) 1978-11-24 1981-06-23 Syva Company Macromolecular environment control in specific receptor assays
US4366241A (en) 1980-08-07 1982-12-28 Syva Company Concentrating zone method in heterogeneous immunoassays
KR101780885B1 (ko) * 2013-03-14 2017-10-11 카리부 바이오사이언시스 인코포레이티드 핵산-표적화 핵산의 조성물 및 방법
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
EP3365357B1 (en) 2015-10-23 2024-02-14 President and Fellows of Harvard College Evolved cas9 proteins for gene editing
AU2017306676B2 (en) * 2016-08-03 2024-02-22 President And Fellows Of Harvard College Adenosine nucleobase editors and uses thereof
WO2018149915A1 (en) * 2017-02-15 2018-08-23 Keygene N.V. Methods of targeted genetic alteration in plant cells
WO2018152197A1 (en) * 2017-02-15 2018-08-23 Massachusetts Institute Of Technology Dna writers, molecular recorders and uses thereof
IL306092A (en) * 2017-03-23 2023-11-01 Harvard College Nucleic base editors that include nucleic acid programmable DNA binding proteins

Also Published As

Publication number Publication date
BR112021007123A2 (pt) 2021-08-10
EA202191033A1 (ru) 2021-12-21
CO2021006336A2 (es) 2021-10-29
CA3116555A1 (en) 2020-04-23
MX2021004301A (es) 2021-08-05
WO2020081568A1 (en) 2020-04-23
US20220290113A1 (en) 2022-09-15
EP3867367A1 (en) 2021-08-25
KR20210077732A (ko) 2021-06-25
AU2019362874A1 (en) 2021-05-27
IL282267A (en) 2021-05-31
CN113166743A (zh) 2021-07-23
JP2022508716A (ja) 2022-01-19

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