BR112020009858A2 - edição de genes com o uso de um dna modificado com extremidades fechadas (cedna) - Google Patents

edição de genes com o uso de um dna modificado com extremidades fechadas (cedna) Download PDF

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Publication number
BR112020009858A2
BR112020009858A2 BR112020009858-2A BR112020009858A BR112020009858A2 BR 112020009858 A2 BR112020009858 A2 BR 112020009858A2 BR 112020009858 A BR112020009858 A BR 112020009858A BR 112020009858 A2 BR112020009858 A2 BR 112020009858A2
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Brazil
Prior art keywords
fact
sequence
cedna vector
cedna
itr
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BR112020009858-2A
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English (en)
Portuguese (pt)
Inventor
Robert M. Kotin
Douglas Kerr
Phillip Samayoa
Ozan Alkan
Matthew J. Simmons
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Generation Bio Co.
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Publication of BR112020009858A2 publication Critical patent/BR112020009858A2/pt

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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
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0016—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
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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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/64—General methods for preparing the vector, for introducing it into the cell or for selecting the vector-containing host
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/66—General methods for inserting a gene into a vector to form a recombinant vector using cleavage and ligation; Use of non-functional linkers or adaptors, e.g. linkers containing the sequence for a restriction endonuclease
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/63—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
    • C—CHEMISTRY; METALLURGY
    • 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)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
    • 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 [CRISPR]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/14011—Baculoviridae
    • C12N2710/14041—Use of virus, viral particle or viral elements as a vector
    • 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
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
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  • Crystallography & Structural Chemistry (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Cell Biology (AREA)
  • Virology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Apparatus Associated With Microorganisms And Enzymes (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
BR112020009858-2A 2017-12-06 2018-12-06 edição de genes com o uso de um dna modificado com extremidades fechadas (cedna) BR112020009858A2 (pt)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762595328P 2017-12-06 2017-12-06
US62/595,328 2017-12-06
US201762607069P 2017-12-18 2017-12-18
US62/607,069 2017-12-18
PCT/US2018/064242 WO2019113310A1 (en) 2017-12-06 2018-12-06 Gene editing using a modified closed-ended dna (cedna)

Publications (1)

Publication Number Publication Date
BR112020009858A2 true BR112020009858A2 (pt) 2020-11-17

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BR112020009858-2A BR112020009858A2 (pt) 2017-12-06 2018-12-06 edição de genes com o uso de um dna modificado com extremidades fechadas (cedna)

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US (1) US20220290186A1 (enExample)
EP (1) EP3720952A4 (enExample)
JP (2) JP2021505159A (enExample)
KR (1) KR20200093635A (enExample)
CN (1) CN111527200A (enExample)
AU (1) AU2018378672A1 (enExample)
BR (1) BR112020009858A2 (enExample)
CA (1) CA3084185A1 (enExample)
IL (1) IL274845A (enExample)
MA (1) MA51113A (enExample)
MX (1) MX2020005808A (enExample)
PH (1) PH12020550771A1 (enExample)
SG (2) SG11202005281XA (enExample)
WO (1) WO2019113310A1 (enExample)

Families Citing this family (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
WO2015105955A1 (en) 2014-01-08 2015-07-16 Flodesign Sonics, Inc. Acoustophoresis device with dual acoustophoretic chamber
US11708572B2 (en) 2015-04-29 2023-07-25 Flodesign Sonics, Inc. Acoustic cell separation techniques and processes
US11377651B2 (en) 2016-10-19 2022-07-05 Flodesign Sonics, Inc. Cell therapy processes utilizing acoustophoresis
NZ762100A (en) 2017-08-09 2025-12-19 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof
EP3690046A2 (en) * 2017-09-28 2020-08-05 Toolgen Incorporated Composition for treatment of hemophilia, comprising crispr/cas system having coagulation factor viii gene inversion correction potential
KR20200120649A (ko) * 2018-02-14 2020-10-21 제너레이션 바이오 컴퍼니 비-바이러스 dna 벡터 및 항체 및 융합 단백질 생산을 위한 이의 용도
CN109022389A (zh) * 2018-07-19 2018-12-18 陕西慧康生物科技有限责任公司 一种大肠杆菌表达人艾杜糖醛酸-2-硫酸酯酶的生产方法
EP3833766A1 (en) 2018-08-09 2021-06-16 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
WO2020097417A1 (en) * 2018-11-09 2020-05-14 Generation Bio Co. Modified closed-ended dna (cedna) comprising symmetrical modified inverted terminal repeats
CN113939595A (zh) 2019-06-07 2022-01-14 瑞泽恩制药公司 包括人源化白蛋白基因座的非人动物
EP4022074A4 (en) * 2019-08-27 2023-11-15 The Trustees of Columbia University in the City of New York Engineered exosomes for targeted delivery
WO2021042045A1 (en) * 2019-08-30 2021-03-04 The Trustees Of Columbia University In The City Of New York Methods for scalable gene insertions
EP4054651A1 (en) 2019-11-08 2022-09-14 Regeneron Pharmaceuticals, Inc. Crispr and aav strategies for x-linked juvenile retinoschisis therapy
BR112022017715A2 (pt) 2020-03-04 2022-11-16 Flagship Pioneering Innovations Vi Llc Métodos e composições para modular um genoma
DE102020111571A1 (de) 2020-03-11 2021-09-16 Immatics US, Inc. Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren
MX2022011805A (es) * 2020-03-24 2023-01-11 Generation Bio Co Vectores de adn no virales y usos de estos para expresar agentes terapéuticos con factor ix.
CN116033915A (zh) * 2020-05-13 2023-04-28 利索基因公司 用于治疗gm1神经节苷脂贮积症及其它病症的组合物和方法
US20230190893A1 (en) * 2020-07-14 2023-06-22 The Regents Of The University Of California Compositions and methods for treating an inherited retinal disease
JP2023535632A (ja) 2020-07-27 2023-08-18 アンジャリウム バイオサイエンシズ エージー Ｄｎａ分子の組成物、その作製方法、及びその使用方法
WO2022061014A1 (en) * 2020-09-16 2022-03-24 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing fviii therapeutics
JP2023542131A (ja) * 2020-09-16 2023-10-05 ジェネレーション バイオ カンパニー フェニルアラニンヒドロキシラーゼ（ｐａｈ）を発現させるための閉端ｄｎａベクター及びその使用
US20250270545A1 (en) * 2020-09-29 2025-08-28 Albert-Ludwigs-Universitat Freiburg Rescue of recombinant adenoviruses by crispr/cas-mediated in vivo terminal resolution
CN112481262B (zh) * 2020-12-04 2022-08-19 中国农业科学院农业基因组研究所 一种基于CRISPR/Cas9基因编辑技术分析增强子细胞生物学功能的方法
CN112852880B (zh) * 2021-01-28 2022-12-06 中吉智药(南京)生物技术有限公司 一种基于诱导型昆虫细胞生产aav基因药物的方法
AU2022216614A1 (en) 2021-02-05 2023-02-23 Christiana Care Gene Editing Institute, Inc. Methods of and compositions for reducing gene expression and/or activity
AU2022237643A1 (en) * 2021-03-19 2023-09-28 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing pfic therapeutics
EP4330416A4 (en) * 2021-04-26 2025-12-03 Univ Massachusetts RAAV-mediated direct in vivo gene editing of hematopoietic stem cells
EP4337177A1 (en) * 2021-05-11 2024-03-20 Modernatx, Inc. Non-viral delivery of dna for prolonged polypeptide expression in vivo
CN117729926A (zh) * 2021-05-28 2024-03-19 比姆医疗股份有限公司 用于使碱基编辑器自失活的组合物和方法
US20240307557A1 (en) * 2021-07-09 2024-09-19 The Board Of Trustees Of The University Of Illinois Therapeutic gene silencing with crispr-cas13
AU2022332276A1 (en) * 2021-08-23 2024-04-04 Bioverativ Therapeutics Inc. Optimized factor viii genes
IL311225A (en) 2021-09-08 2024-05-01 Flagship Pioneering Innovations Vi Llc Methods and compositions for genome modulation
IL312184A (en) 2021-10-18 2024-06-01 Flagship Pioneering Innovations Vii Llc Dna compositions and related methods
CN118541475A (zh) * 2021-12-27 2024-08-23 亘喜生物科技（上海）有限公司 用于细胞修饰的系统和方法
WO2024040222A1 (en) 2022-08-19 2024-02-22 Generation Bio Co. Cleavable closed-ended dna (cedna) and methods of use thereof
EP4587454A2 (en) * 2022-09-16 2025-07-23 Joseph Fenton Lawler Trans-splicing methods and compositions for generation of single sex offspring
WO2024229153A2 (en) * 2023-05-01 2024-11-07 Seattle Children's Hospital D/B/A Seattle Children's Research Institute Systems and methods to provide therapeutic factor viii using ultrasound-mediated nucleic acid delivery
WO2025064507A1 (en) * 2023-09-19 2025-03-27 Poseida Therapeutics, Inc. Compositions and methods for integration of viral vectors
WO2025085119A2 (en) * 2023-10-17 2025-04-24 Generation Bio Co. Synthetic partially single-stranded nucleic acid compositions and uses and methods therefor
WO2025083617A1 (en) 2023-10-18 2025-04-24 Nanocell Therapeutics Holdings B.V. Methods and compositions for nucleic construct delivery
WO2025106523A1 (en) * 2023-11-14 2025-05-22 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of f8 expression
WO2025106834A1 (en) * 2023-11-17 2025-05-22 Temple University-Of The Commonwealth System Of Higher Education A single-plasmid system for enhanced crispr-cas9 mediated homologous recombination

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2500434A1 (en) * 2011-03-12 2012-09-19 Association Institut de Myologie Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
US10604771B2 (en) * 2013-05-10 2020-03-31 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
MX374529B (es) * 2013-12-12 2025-03-06 Broad Inst Inc Suministro, uso y aplicaciones terapeuticas de sistemas y composiciones crispr-cas para edicion del genoma.
WO2015138620A1 (en) * 2014-03-11 2015-09-17 University Of Washington Restricting nuclear protein to specific phases of the cell cycle
US20180112213A1 (en) * 2015-03-25 2018-04-26 Editas Medicine, Inc. Crispr/cas-related methods, compositions and components
WO2016176191A1 (en) * 2015-04-27 2016-11-03 The Trustees Of The University Of Pennsylvania Dual aav vector system for crispr/cas9 mediated correction of human disease
WO2016205728A1 (en) * 2015-06-17 2016-12-22 Massachusetts Institute Of Technology Crispr mediated recording of cellular events
KR102336362B1 (ko) * 2016-03-03 2021-12-08 보이저 테라퓨틱스, 인크. 비-바이러스 유전자 전달을 위한 폐쇄형-말단 선형 듀플렉스 dna
CN111132699A (zh) * 2017-09-08 2020-05-08 世代生物公司 修饰的封闭端dna(cedna)

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MX2020005808A (es) 2020-10-28
SG10202012132WA (en) 2021-01-28
SG11202005281XA (en) 2020-07-29
AU2018378672A1 (en) 2020-07-09
JP2024003220A (ja) 2024-01-11
CA3084185A1 (en) 2019-06-13
US20220290186A1 (en) 2022-09-15
EP3720952A1 (en) 2020-10-14
PH12020550771A1 (en) 2021-05-10
JP2021505159A (ja) 2021-02-18
IL274845A (en) 2020-07-30
RU2020121128A (ru) 2022-01-11
WO2019113310A1 (en) 2019-06-13
KR20200093635A (ko) 2020-08-05
MA51113A (fr) 2020-10-14
EP3720952A4 (en) 2021-09-01
CN111527200A (zh) 2020-08-11

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