JP7817176B2 - 代謝性肝障害の治療のための組成物と方法 - Google Patents

代謝性肝障害の治療のための組成物と方法

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Publication number
JP7817176B2
JP7817176B2 JP2022552651A JP2022552651A JP7817176B2 JP 7817176 B2 JP7817176 B2 JP 7817176B2 JP 2022552651 A JP2022552651 A JP 2022552651A JP 2022552651 A JP2022552651 A JP 2022552651A JP 7817176 B2 JP7817176 B2 JP 7817176B2
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nucleic acid
seq
acid sequence
polynucleotide
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チアン チンチン
ダウン ジュリアン
リー シアンホン
エム．オスタータグ エリック
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ポセイダ セラピューティクス，インコーポレイティド
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    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • 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
    • 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
    • C12N9/10—Transferases (2.)
    • C12N9/1003—Transferases (2.) transferring one-carbon groups (2.1)
    • C12N9/1018—Carboxy- and carbamoyl transferases (2.1.3)
    • 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
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241—Nucleotidyltransferases (2.7.7)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/052—Animals comprising random inserted nucleic acids (transgenic) inducing gain of function
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • 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
    • 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
    • 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
    • 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
    • C12N2800/90—Vectors containing a transposable element
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/40—Vector systems having a special element relevant for transcription being an insulator
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y201/00—Transferases transferring one-carbon groups (2.1)
    • C12Y201/03—Carboxy- and carbamoyltransferases (2.1.3)
    • C12Y201/03003—Ornithine carbamoyltransferase (2.1.3.3)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/07—Nucleotidyltransferases (2.7.7)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2022552651A 2020-03-04 2021-03-04 代謝性肝障害の治療のための組成物と方法 Active JP7817176B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202062985047P 2020-03-04 2020-03-04
US62/985,047 2020-03-04
US202063121488P 2020-12-04 2020-12-04
US63/121,488 2020-12-04
PCT/US2021/020929 WO2021178707A1 (en) 2020-03-04 2021-03-04 Compositions and methods for the treatment of metabolic liver disorders

Publications (4)

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JP2023515692A JP2023515692A (ja) 2023-04-13
JPWO2021178707A5 JPWO2021178707A5 (https=) 2024-03-12
JP2023515692A5 JP2023515692A5 (https=) 2024-03-12
JP7817176B2 true JP7817176B2 (ja) 2026-02-18

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US (1) US12540335B2 (https=)
EP (1) EP4114469A1 (https=)
JP (1) JP7817176B2 (https=)
KR (1) KR20220149588A (https=)
CN (1) CN115515646A (https=)
AU (1) AU2021230361A1 (https=)
CA (1) CA3169529A1 (https=)
WO (1) WO2021178707A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022061002A1 (en) * 2020-09-18 2022-03-24 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Novel adeno-associated viral (aav) vectors to treat hereditary methylmalonic acidemia (mma) caused by methylmalonyl-coa mutase (mmut) deficiency
CA3094859A1 (en) * 2020-10-01 2022-04-01 Entos Pharmaceuticals Inc. Proteolipid vesicles formulated with fusion associated small transmembrane proteins
WO2022187671A1 (en) * 2021-03-04 2022-09-09 Poseida Therapeutics, Inc. Compositions and methods for the treatment of hemophilia
GB202205606D0 (en) * 2022-04-14 2022-06-01 Genespire S R L Lentiviral vector
AU2024224841A1 (en) * 2023-02-21 2025-08-21 Poseida Therapeutics, Inc. Aav piggybac transposon polynucleotide compositions and methods of use therefor
KR20250173518A (ko) * 2023-04-05 2025-12-10 포세이다 테라퓨틱스, 인크. 트랜스포사제 폴리뉴클레오티드 및 그의 용도
IL325133A (en) * 2023-06-07 2026-02-01 Res Inst Nationwide Childrens Hospital Gene therapy for lysosomal acid lipase deficiency (LAL-D)
WO2025171163A1 (en) * 2024-02-06 2025-08-14 Duke University Compositions for and methods of treating lysosomal storage diseases

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011126808A2 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
WO2014003553A1 (en) 2012-06-27 2014-01-03 Arthrogen B.V. Combination for treating an inflammatory disorder
WO2015139093A1 (en) 2014-03-21 2015-09-24 The Sydney Children's Hospitals Network (Randwick And Westmead) (Incorporating The Royal Alexandra Hospital For Children) Stable gene transfer to proliferating cells
WO2017021359A1 (en) 2015-08-03 2017-02-09 Myodopa Limited Systemic synthesis and regulation of l-dopa
WO2019173636A1 (en) 2018-03-07 2019-09-12 Poseida Therapeutics, Inc. Cartyrin compositions and methods for use

Family Cites Families (51)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3773919A (en) 1969-10-23 1973-11-20 Du Pont Polylactide-drug mixtures
US4309989A (en) 1976-02-09 1982-01-12 The Curators Of The University Of Missouri Topical application of medication by ultrasound with coupling agent
FR2374910A1 (fr) 1976-10-23 1978-07-21 Choay Sa Preparation a base d'heparine, comprenant des liposomes, procede pour l'obtenir et medicaments contenant de telles preparations
US4554101A (en) 1981-01-09 1985-11-19 New York Blood Center, Inc. Identification and preparation of epitopes on antigens and allergens on the basis of hydrophilicity
US4656134A (en) 1982-01-11 1987-04-07 Board Of Trustees Of Leland Stanford Jr. University Gene amplification in eukaryotic cells
US5168062A (en) 1985-01-30 1992-12-01 University Of Iowa Research Foundation Transfer vectors and microorganisms containing human cytomegalovirus immediate-early promoter-regulatory DNA sequence
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US4965188A (en) 1986-08-22 1990-10-23 Cetus Corporation Process for amplifying, detecting, and/or cloning nucleic acid sequences using a thermostable enzyme
US4766067A (en) 1985-05-31 1988-08-23 President And Fellows Of Harvard College Gene amplification
GB8601597D0 (en) 1986-01-23 1986-02-26 Wilson R H Nucleotide sequences
US4800159A (en) 1986-02-07 1989-01-24 Cetus Corporation Process for amplifying, detecting, and/or cloning nucleic acid sequences
US4767402A (en) 1986-07-08 1988-08-30 Massachusetts Institute Of Technology Ultrasound enhancement of transdermal drug delivery
EP0545913B1 (en) 1986-08-18 1999-02-24 Emisphere Technologies, Inc. Delivery systems for pharmacological agents
US4889818A (en) 1986-08-22 1989-12-26 Cetus Corporation Purified thermostable enzyme
US4921794A (en) 1987-01-14 1990-05-01 President And Fellows Of Harvard College T7 DNA polymerase
US4795699A (en) 1987-01-14 1989-01-03 President And Fellows Of Harvard College T7 DNA polymerase
US5130238A (en) 1988-06-24 1992-07-14 Cangene Corporation Enhanced nucleic acid amplification process
US5091310A (en) 1988-09-23 1992-02-25 Cetus Corporation Structure-independent dna amplification by the polymerase chain reaction
US5066584A (en) 1988-09-23 1991-11-19 Cetus Corporation Methods for generating single stranded dna by the polymerase chain reaction
US5142033A (en) 1988-09-23 1992-08-25 Hoffmann-La Roche Inc. Structure-independent DNA amplification by the polymerase chain reaction
US4994370A (en) 1989-01-03 1991-02-19 The United States Of America As Represented By The Department Of Health And Human Services DNA amplification technique
US5266491A (en) 1989-03-14 1993-11-30 Mochida Pharmaceutical Co., Ltd. DNA fragment and expression plasmid containing the DNA fragment
US5580575A (en) 1989-12-22 1996-12-03 Imarx Pharmaceutical Corp. Therapeutic drug delivery systems
US5580734A (en) 1990-07-13 1996-12-03 Transkaryotic Therapies, Inc. Method of producing a physical map contigous DNA sequences
US5733761A (en) 1991-11-05 1998-03-31 Transkaryotic Therapies, Inc. Protein production and protein delivery
US5641670A (en) 1991-11-05 1997-06-24 Transkaryotic Therapies, Inc. Protein production and protein delivery
US5643252A (en) 1992-10-28 1997-07-01 Venisect, Inc. Laser perforator
US5849695A (en) 1993-01-13 1998-12-15 The Regents Of The University Of California Parathyroid hormone analogues useful for treatment of osteoporosis and disorders of calcium meatabolism in mammals
CZ206195A3 (en) 1993-02-12 1996-04-17 Univ Leland Stanford Junior Controlled transcription of target genes and other biological materials
US5514670A (en) 1993-08-13 1996-05-07 Pharmos Corporation Submicron emulsions for delivery of peptides
US5814599A (en) 1995-08-04 1998-09-29 Massachusetts Insitiute Of Technology Transdermal delivery of encapsulated drugs
US5730723A (en) 1995-10-10 1998-03-24 Visionary Medical Products Corporation, Inc. Gas pressured needle-less injection device and method
US6218185B1 (en) 1996-04-19 2001-04-17 The United States Of America As Represented By The Secretary Of Agriculture Piggybac transposon-based genetic transformation system for insects
US5827729A (en) 1996-04-23 1998-10-27 Advanced Tissue Sciences Diffusion gradient bioreactor and extracorporeal liver device using a three-dimensional liver tissue
US5879681A (en) 1997-02-07 1999-03-09 Emisphere Technolgies Inc. Compounds and compositions for delivering active agents
IL120943A (en) 1997-05-29 2004-03-28 Univ Ben Gurion A system for administering drugs through the skin
US6309663B1 (en) 1999-08-17 2001-10-30 Lipocine Inc. Triglyceride-free compositions and methods for enhanced absorption of hydrophilic therapeutic agents
US6962810B2 (en) 2000-10-31 2005-11-08 University Of Notre Dame Du Lac Methods and compositions for transposition using minimal segments of the eukaryotic transformation vector piggyBac
EP2160461B1 (en) 2007-07-04 2012-08-08 Max-Delbrück-Centrum für Molekulare Medizin (MDC) Hyperactive variants of the transposase protein of the transposon system sleeping beauty
WO2010099296A1 (en) 2009-02-26 2010-09-02 Transposagen Biopharmaceuticals, Inc. Hyperactive piggybac transposases
US20120156170A1 (en) * 2009-08-31 2012-06-21 Tadashi Tanabe Drug composition for angiogenesis therapy
WO2012050919A2 (en) * 2010-09-28 2012-04-19 The University Of Notre Dame Chimeric spider silk and uses thereof
WO2012074758A1 (en) * 2010-11-16 2012-06-07 Transposagen Bioharmaceuticals, Inc. Hyperactive piggybac transposases
WO2014143884A2 (en) 2013-03-15 2014-09-18 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Synthetic methylmalonyl-coa mutase transgene for the treatment of mut class methylmalonic acidemia (mma)
RS60902B1 (sr) 2014-03-09 2020-11-30 Univ Pennsylvania Kompozicije korisne u lečenju nedostatka ornitinske transkarbamilaze (otc)
US10927384B2 (en) 2014-04-09 2021-02-23 Dna Twopointo Inc. DNA vectors, transposons and transposases for eukaryotic genome modification
US20170112108A1 (en) * 2015-10-22 2017-04-27 Hera Testing Laboratories, Inc. Genetically modified non-human mammals having modified liver cells and/or tissue and methods of making same
WO2017099838A1 (en) 2015-12-11 2017-06-15 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Gene therapy for combined methylmalonic acidemia/aciduria and hyperhomocysteinemia/homocystinuria, cobalamin c type, and deficiency of mmachc
EP3680280A4 (en) 2017-09-05 2021-06-02 Toray Industries, Inc. FIBER REINFORCED THERMOPLASTIC RESIN MOLDINGS
EP3847197A1 (en) 2018-09-05 2021-07-14 Poseida Therapeutics, Inc. Allogeneic cell compositions and methods of use

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011126808A2 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
WO2014003553A1 (en) 2012-06-27 2014-01-03 Arthrogen B.V. Combination for treating an inflammatory disorder
WO2015139093A1 (en) 2014-03-21 2015-09-24 The Sydney Children's Hospitals Network (Randwick And Westmead) (Incorporating The Royal Alexandra Hospital For Children) Stable gene transfer to proliferating cells
WO2017021359A1 (en) 2015-08-03 2017-02-09 Myodopa Limited Systemic synthesis and regulation of l-dopa
WO2019173636A1 (en) 2018-03-07 2019-09-12 Poseida Therapeutics, Inc. Cartyrin compositions and methods for use

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Definition:ornithine carbamoyltransferase, mitochondrial precursor，Database: DDBJ/EMBL/GenBank [online], Accession No. NP_000522，02-JAN-2020 uploaded, [retrieved on 2025-02-20], <https://www.ncbi.nlm.nih.gov/protein/38788445?sat=48&satkey=77516498>
JHEP Reports，2020年，Vol.2, No.100065，pp.1-11，available online 27 December 2019

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JP2023515692A (ja) 2023-04-13
WO2021178707A1 (en) 2021-09-10
AU2021230361A1 (en) 2022-09-08
CA3169529A1 (en) 2021-09-10
US12540335B2 (en) 2026-02-03
US20230104091A1 (en) 2023-04-06
EP4114469A1 (en) 2023-01-11
CN115515646A (zh) 2022-12-23
KR20220149588A (ko) 2022-11-08

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