EA202191033A1 - Программируемое редактирование оснований днк посредством слитых белков nme2cas9-дезаминаза - Google Patents
Программируемое редактирование оснований днк посредством слитых белков nme2cas9-дезаминазаInfo
- Publication number
- EA202191033A1 EA202191033A1 EA202191033A EA202191033A EA202191033A1 EA 202191033 A1 EA202191033 A1 EA 202191033A1 EA 202191033 A EA202191033 A EA 202191033A EA 202191033 A EA202191033 A EA 202191033A EA 202191033 A1 EA202191033 A1 EA 202191033A1
- Authority
- EA
- Eurasian Patent Office
- Prior art keywords
- editing
- nme2cas9
- fusion proteins
- single nucleotide
- dna base
- Prior art date
Links
Classifications
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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
- 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 RNAses, DNAses
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
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- C—CHEMISTRY; METALLURGY
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- 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
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- 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
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- 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
- C12N15/62—DNA sequences coding for fusion proteins
- C12N15/625—DNA sequences coding for fusion proteins containing a sequence coding for a signal sequence
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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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
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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
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
- C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
- C12Y305/04004—Adenosine deaminase (3.5.4.4)
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
- C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
- C12Y305/04005—Cytidine deaminase (3.5.4.5)
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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 [CRISPRs]
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14171—Demonstrated in vivo effect
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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
- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- Cell Biology (AREA)
- Diabetes (AREA)
- Veterinary Medicine (AREA)
- Mycology (AREA)
- Virology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
Abstract
Настоящее изобретение относится к области редактирования генов. В частности, редактирование генов направлено на редактирование единичных нуклеотидных оснований. Например, такое редактирование единичных нуклеотидных оснований приводит к преобразованию пары оснований C-G в пару оснований T-A. Высокая точность и аккуратность описанного в настоящем описании однонуклеотидного редактора достигается посредством нуклеазы NmeCas9, которая слита с белком нуклеотиддезаминазой. Компактная природа NmeCas9, сопряженной с более высоким количеством совместимых прилегающих к протоспейсеру мотивов, обеспечивает наличие у слитых конструкций Cas9, описанных в настоящем описании, окна редактирования генов, которое может редактировать участки, на которые не нацеливаются другие общепринятые редактирующие основания платформы SpyCas9.
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 |
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EA202191033A1 true EA202191033A1 (ru) | 2021-12-21 |
Family
ID=68621349
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
EA202191033A EA202191033A1 (ru) | 2018-10-15 | 2019-10-15 | Программируемое редактирование оснований днк посредством слитых белков nme2cas9-дезаминаза |
Country Status (14)
Country | Link |
---|---|
US (1) | US20220290113A1 (ru) |
EP (1) | EP3867367A1 (ru) |
JP (2) | JP2022508716A (ru) |
KR (1) | KR20210077732A (ru) |
CN (1) | CN113166743A (ru) |
AU (1) | AU2019362874A1 (ru) |
BR (1) | BR112021007123A2 (ru) |
CA (1) | CA3116555A1 (ru) |
CO (1) | CO2021006336A2 (ru) |
EA (1) | EA202191033A1 (ru) |
IL (1) | IL282267A (ru) |
MX (1) | MX2021004301A (ru) |
SG (1) | SG11202103722TA (ru) |
WO (1) | WO2020081568A1 (ru) |
Families Citing this family (18)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
JP2023525304A (ja) | 2020-05-08 | 2023-06-15 | ザ ブロード インスティテュート,インコーポレーテッド | 標的二本鎖ヌクレオチド配列の両鎖同時編集のための方法および組成物 |
GB202010348D0 (en) | 2020-07-06 | 2020-08-19 | Univ Wageningen | Base editing tools |
CA3204997A1 (en) | 2020-12-11 | 2022-06-16 | Intellia Therapeutics, Inc. | Compositions and methods for reducing mhc class ii in a cell |
JP2023553935A (ja) | 2020-12-11 | 2023-12-26 | インテリア セラピューティクス,インコーポレイテッド | 脱アミノ化を伴うゲノム編集のためのポリヌクレオチド、組成物、及び方法 |
CA3206284A1 (en) | 2020-12-23 | 2022-06-30 | Intellia Therapeutics, Inc. | Compositions and methods for reducing hla-a in a cell |
CA3205042A1 (en) | 2020-12-23 | 2022-06-30 | Intellia Therapeutics, Inc. | Compositions and methods for genetically modifying ciita in a cell |
CA3236778A1 (en) * | 2021-11-02 | 2023-05-11 | Erik SONTHEIMER | Nme2cas9 inlaid domain fusion proteins |
IL312508A (en) | 2021-11-03 | 2024-07-01 | Intellia Therapeutics Inc | Polynucleotides, compounds and methods for genome editing |
AU2023248451A1 (en) * | 2022-04-04 | 2024-10-17 | President And Fellows Of Harvard College | 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 |
WO2023245108A2 (en) | 2022-06-16 | 2023-12-21 | Intellia Therapeutics, Inc. | Compositions and methods for reducing mhc class i in a cell |
TW202413631A (zh) | 2022-06-16 | 2024-04-01 | 美商英特利亞醫療公司 | 用於基因體編輯之組成物及方法 |
TW202408595A (zh) | 2022-06-16 | 2024-03-01 | 美商英特利亞醫療公司 | 用於對細胞進行遺傳修飾之方法及組合物 |
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 |
WO2024138189A2 (en) | 2022-12-22 | 2024-06-27 | Intellia Therapeutics, Inc. | Methods for analyzing nucleic acid cargos of lipid nucleic acid assemblies |
WO2024186890A1 (en) | 2023-03-06 | 2024-09-12 | Intellia Therapeutics, Inc. | Compositions and methods for hepatitis b virus (hbv) genome editing |
Family Cites Families (14)
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 |
US4275149A (en) | 1978-11-24 | 1981-06-23 | Syva Company | Macromolecular environment control in specific receptor assays |
US4277437A (en) | 1978-04-05 | 1981-07-07 | Syva Company | Kit for carrying out chemically induced fluorescence immunoassay |
US4366241A (en) | 1980-08-07 | 1982-12-28 | Syva Company | Concentrating zone method in heterogeneous immunoassays |
CN105980575A (zh) * | 2013-03-14 | 2016-09-28 | 卡里布生物科学公司 | 以核酸为靶的核酸的组合物和方法 |
US9840699B2 (en) | 2013-12-12 | 2017-12-12 | President And Fellows Of Harvard College | Methods for nucleic acid editing |
US10167457B2 (en) * | 2015-10-23 | 2019-01-01 | President And Fellows Of Harvard College | Nucleobase editors and uses thereof |
SG11201900907YA (en) * | 2016-08-03 | 2019-02-27 | Harvard College | Adenosine nucleobase editors and uses thereof |
US20200063127A1 (en) * | 2017-02-15 | 2020-02-27 | Massachusetts Institute Of Technology | Dna writers, molecular recorders and uses thereof |
KR20190117536A (ko) * | 2017-02-15 | 2019-10-16 | 키진 엔.브이. | 식물 세포에서 표적 유전적 변형 방법 |
US11268082B2 (en) * | 2017-03-23 | 2022-03-08 | President And Fellows Of Harvard College | Nucleobase editors comprising nucleic acid programmable DNA binding proteins |
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2019
- 2019-10-15 SG SG11202103722TA patent/SG11202103722TA/en unknown
- 2019-10-15 KR KR1020217014669A patent/KR20210077732A/ko unknown
- 2019-10-15 CN CN201980077403.7A patent/CN113166743A/zh active Pending
- 2019-10-15 BR BR112021007123-7A patent/BR112021007123A2/pt unknown
- 2019-10-15 MX MX2021004301A patent/MX2021004301A/es unknown
- 2019-10-15 WO PCT/US2019/056341 patent/WO2020081568A1/en active Application Filing
- 2019-10-15 EA EA202191033A patent/EA202191033A1/ru unknown
- 2019-10-15 EP EP19806344.8A patent/EP3867367A1/en active Pending
- 2019-10-15 JP JP2021545287A patent/JP2022508716A/ja active Pending
- 2019-10-15 AU AU2019362874A patent/AU2019362874A1/en active Pending
- 2019-10-15 CA CA3116555A patent/CA3116555A1/en active Pending
- 2019-10-15 US US17/285,440 patent/US20220290113A1/en active Pending
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2021
- 2021-04-12 IL IL282267A patent/IL282267A/en unknown
- 2021-05-14 CO CONC2021/0006336A patent/CO2021006336A2/es unknown
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2024
- 2024-07-03 JP JP2024107552A patent/JP2024121000A/ja active Pending
Also Published As
Publication number | Publication date |
---|---|
US20220290113A1 (en) | 2022-09-15 |
WO2020081568A1 (en) | 2020-04-23 |
CN113166743A (zh) | 2021-07-23 |
CO2021006336A2 (es) | 2021-10-29 |
MX2021004301A (es) | 2021-08-05 |
SG11202103722TA (en) | 2021-05-28 |
KR20210077732A (ko) | 2021-06-25 |
CA3116555A1 (en) | 2020-04-23 |
EP3867367A1 (en) | 2021-08-25 |
AU2019362874A1 (en) | 2021-05-27 |
JP2022508716A (ja) | 2022-01-19 |
IL282267A (en) | 2021-05-31 |
BR112021007123A2 (pt) | 2021-08-10 |
JP2024121000A (ja) | 2024-09-05 |
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