RS58862B1 - Rnki indukovana supresija hantingtonovog gena - Google Patents
Rnki indukovana supresija hantingtonovog genaInfo
- Publication number
- RS58862B1 RS58862B1 RS20190744A RSP20190744A RS58862B1 RS 58862 B1 RS58862 B1 RS 58862B1 RS 20190744 A RS20190744 A RS 20190744A RS P20190744 A RSP20190744 A RS P20190744A RS 58862 B1 RS58862 B1 RS 58862B1
- Authority
- RS
- Serbia
- Prior art keywords
- gene suppression
- huntingtin gene
- rnai induced
- induced huntingtin
- rnai
- Prior art date
Links
Classifications
-
- 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
- C12N15/11—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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
- C12N2310/141—MicroRNAs, miRNAs
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3519—Fusion with another nucleic acid
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
-
- 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
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted vectors
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP14200308 | 2014-12-24 | ||
| PCT/EP2015/081157 WO2016102664A1 (en) | 2014-12-24 | 2015-12-23 | Rnai induced huntingtin gene suppression |
| EP15817385.6A EP3237618B1 (en) | 2014-12-24 | 2015-12-23 | Rnai induced huntingtin gene suppression |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| RS58862B1 true RS58862B1 (sr) | 2019-07-31 |
Family
ID=52282540
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RS20190744A RS58862B1 (sr) | 2014-12-24 | 2015-12-23 | Rnki indukovana supresija hantingtonovog gena |
Country Status (25)
| Country | Link |
|---|---|
| US (4) | US10174321B2 (sr) |
| EP (2) | EP3237618B1 (sr) |
| JP (2) | JP6839094B2 (sr) |
| KR (1) | KR102636633B1 (sr) |
| CN (1) | CN108064292B (sr) |
| AU (1) | AU2015370903B2 (sr) |
| BR (1) | BR112017013573A2 (sr) |
| CA (1) | CA2971920A1 (sr) |
| CO (1) | CO2017007335A2 (sr) |
| CY (1) | CY1121733T1 (sr) |
| DK (1) | DK3237618T3 (sr) |
| EA (1) | EA037696B1 (sr) |
| ES (1) | ES2732023T3 (sr) |
| HK (1) | HK1246344B (sr) |
| HR (1) | HRP20190992T1 (sr) |
| HU (1) | HUE043842T2 (sr) |
| IL (1) | IL252990B (sr) |
| LT (1) | LT3237618T (sr) |
| MX (1) | MX2017008500A (sr) |
| PL (1) | PL3237618T3 (sr) |
| PT (1) | PT3237618T (sr) |
| RS (1) | RS58862B1 (sr) |
| SI (1) | SI3237618T1 (sr) |
| TR (1) | TR201908969T4 (sr) |
| WO (1) | WO2016102664A1 (sr) |
Families Citing this family (54)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR101881596B1 (ko) | 2008-12-02 | 2018-07-24 | 웨이브 라이프 사이언시스 재팬 인코포레이티드 | 인 원자 변형된 핵산의 합성 방법 |
| SG177564A1 (en) | 2009-07-06 | 2012-02-28 | Ontorii Inc | Novel nucleic acid prodrugs and methods of use thereof |
| WO2012039448A1 (ja) | 2010-09-24 | 2012-03-29 | 株式会社キラルジェン | 不斉補助基 |
| EP2734208B1 (en) | 2011-07-19 | 2017-03-01 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
| WO2014010718A1 (ja) | 2012-07-13 | 2014-01-16 | 株式会社新日本科学 | キラル核酸アジュバント |
| KR102213609B1 (ko) | 2012-07-13 | 2021-02-08 | 웨이브 라이프 사이언시스 리미티드 | 키랄 제어 |
| EP2872485B1 (en) | 2012-07-13 | 2020-12-16 | Wave Life Sciences Ltd. | Asymmetric auxiliary group |
| JPWO2015108048A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤 |
| WO2015108047A1 (ja) | 2014-01-15 | 2015-07-23 | 株式会社新日本科学 | 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤 |
| JPWO2015108046A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 抗アレルギー作用を有するキラル核酸アジュバンド及び抗アレルギー剤 |
| AU2015207773B2 (en) | 2014-01-16 | 2021-06-17 | Wave Life Sciences Ltd. | Chiral design |
| EP3218484A4 (en) | 2014-11-14 | 2018-05-30 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| SG11201703419UA (en) | 2014-11-14 | 2017-05-30 | Voyager Therapeutics Inc | Modulatory polynucleotides |
| PT3237618T (pt) * | 2014-12-24 | 2019-07-04 | Uniqure Ip Bv | Supressão do gene huntingtina induzida por arni |
| JP2018506304A (ja) | 2015-02-10 | 2018-03-08 | ジェンザイム・コーポレーション | バリアントＲＮＡｉ |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| LT3386511T (lt) | 2015-12-10 | 2021-08-25 | Ptc Therapeutics, Inc. | Hantingtono ligos gydymo būdai |
| MA45270A (fr) | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| WO2017201258A1 (en) | 2016-05-18 | 2017-11-23 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
| EP3458588A4 (en) | 2016-05-18 | 2020-01-15 | Voyager Therapeutics, Inc. | MODULATING POLYNUCLEOTIDES |
| US10457940B2 (en) * | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
| WO2018204803A1 (en) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
| EP3634953B1 (en) | 2017-06-05 | 2024-01-03 | PTC Therapeutics, Inc. | Compounds for treating huntington's disease |
| CN111182898B (zh) | 2017-06-28 | 2024-04-16 | Ptc医疗公司 | 用于治疗亨廷顿氏病的方法 |
| EP3645121A4 (en) | 2017-06-28 | 2021-03-17 | PTC Therapeutics, Inc. | HUNTINGTON'S DISEASE TREATMENT METHODS |
| CA3076191A1 (en) * | 2017-09-22 | 2019-03-28 | Genzyme Corporation | Variant rnai |
| AU2018352236A1 (en) | 2017-10-16 | 2020-04-23 | The Curators Of The University Of Missouri | Treatment of amyotrophic lateral sclerosis (ALS) |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| CN108384803A (zh) * | 2017-12-19 | 2018-08-10 | 上海百力格生物技术有限公司 | 一种用于miRNA干扰载体构建筛选特异对照的质粒及其构建方法 |
| US20210009590A1 (en) | 2018-03-27 | 2021-01-14 | Ptc Therapeutics, Inc. | Compounds for treating huntington's disease |
| US20210269825A1 (en) * | 2018-05-15 | 2021-09-02 | University Of Washington | Compositions and methods for reducing spliceopathy and treating rna dominance disorders |
| EP3794125A4 (en) * | 2018-05-15 | 2022-07-13 | University of Massachusetts | MODIFIED AAV CONSTRUCTS AND USES THEREOF |
| EP3814357B1 (en) | 2018-06-27 | 2024-05-01 | PTC Therapeutics, Inc. | Heterocyclic and heteroaryl compounds for treating huntington's disease |
| JP2021529513A (ja) | 2018-07-02 | 2021-11-04 | ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． | 筋萎縮性側索硬化症および脊髄に関連する障害の治療 |
| EP3850098A1 (en) | 2018-09-12 | 2021-07-21 | uniQure IP B.V. | Rnai induced c9orf72 suppression for the treatment of als/ftd |
| EP3884067A1 (en) | 2018-11-19 | 2021-09-29 | uniQure IP B.V. | A companion diagnostic to monitor the effects of gene therapy |
| WO2020104295A1 (en) | 2018-11-19 | 2020-05-28 | Uniqure Ip B.V. | Rnai induced reduction of ataxin-3 for the treatment of spinocerebellar ataxia type 3 |
| WO2020104469A1 (en) | 2018-11-19 | 2020-05-28 | Uniqure Ip B.V. | Method and means to deliver mirna to target cells |
| JP2022516779A (ja) * | 2019-01-09 | 2022-03-02 | ウニベルシダージ デ コインブラ | 二本鎖ｒｎａ及びその使用 |
| WO2021005223A1 (en) | 2019-07-10 | 2021-01-14 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods for the treatment of epilepsy |
| EP4031148A1 (en) | 2019-09-16 | 2022-07-27 | uniQure IP B.V. | Targeting misspliced transcripts in genetic disorders |
| AR120341A1 (es) * | 2019-11-01 | 2022-02-09 | Alnylam Pharmaceuticals Inc | COMPOSICIONES DE AGENTES DE ARNi CONTRA LA HUNTINGTINA (HTT) Y SUS MÉTODOS DE USO |
| KR20220093335A (ko) | 2019-11-01 | 2022-07-05 | 노파르티스 아게 | 헌팅턴병의 진행을 늦추는 치료를 위한 스플라이싱 조절제의 용도 |
| JP2023521347A (ja) * | 2020-04-07 | 2023-05-24 | ユニキュアー アイピー ビー．ブイ． | アンジオポエチン様３（ａｎｇｐｔｌ３）をサイレンシングするための遺伝子構築物及びその使用 |
| WO2021247995A2 (en) | 2020-06-04 | 2021-12-09 | Voyager Therapeutics, Inc. | Compositions and methods of treating neuropathic pain |
| WO2022031591A2 (en) * | 2020-08-03 | 2022-02-10 | University Of Massachusetts | Oligonucleotides for htt-1a modulation |
| TW202304446A (zh) | 2021-03-29 | 2023-02-01 | 瑞士商諾華公司 | 剪接調節子用於減慢杭丁頓氏舞蹈症進展的治療之用途 |
| JP2024511207A (ja) * | 2021-03-30 | 2024-03-12 | 南京大学 | ハンチントン病を治療するためのｒｎａ送達システム |
| EP4359525A1 (en) | 2021-06-21 | 2024-05-01 | uniQure biopharma B.V. | Gene constructs for silencing alpha-synuclein and uses thereof |
| WO2023147058A2 (en) * | 2022-01-27 | 2023-08-03 | Asklepios Biopharmaceutical, Inc. | Compositions for treating neurological disease |
| WO2023198745A1 (en) | 2022-04-12 | 2023-10-19 | Uniqure Biopharma B.V. | Nucleic acid regulation of apoe |
| WO2023198662A1 (en) | 2022-04-12 | 2023-10-19 | Uniqure Biopharma B.V. | Novel systems for nucleic acid regulation |
| WO2023198702A1 (en) | 2022-04-12 | 2023-10-19 | Uniqure Biopharma B.V. | Nucleic acid regulation of c9orf72 |
| WO2023198663A1 (en) | 2022-04-12 | 2023-10-19 | Uniqure Biopharma B.V. | Nucleic acid regulation of snca |
Family Cites Families (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7589189B2 (en) * | 2003-05-14 | 2009-09-15 | Japan Science And Technology Agency | Inhibition of the expression of huntingtin gene |
| US8680063B2 (en) | 2003-09-12 | 2014-03-25 | University Of Massachusetts | RNA interference for the treatment of gain-of-function disorders |
| WO2008005562A2 (en) | 2006-07-07 | 2008-01-10 | University Of Massachusetts | Rna silencing compositions and methods for the treatment of huntington's disease |
| EP1735443A2 (en) | 2004-04-14 | 2006-12-27 | Sirna Therapeutics, Inc. | RNA INTERFERENCE MEDIATED TREATMENT OF POLYGLUTAMINE (POLYQ) REPEAT EXPANSION DISEASES USING SHORT INTERFERING NUCLEIC ACID (siNA) |
| WO2007046703A2 (en) | 2005-10-20 | 2007-04-26 | Amsterdam Molecular Therapeutics B.V. | Improved aav vectors produced in insect cells |
| EP3023500B1 (en) | 2006-06-21 | 2020-02-12 | uniQure IP B.V. | Insect cells for the production of aav vectors |
| WO2008134646A2 (en) | 2007-04-26 | 2008-11-06 | University Of Iowa Research Foundation | Rna interference suppression of neurodegenerative diseases and methods of use thereof |
| EP2530152B1 (en) | 2007-05-31 | 2017-12-27 | University of Iowa Research Foundation | Reduction of off-target RNA interference toxicity |
| PT2173888T (pt) | 2007-07-26 | 2016-11-17 | Uniqure Ip Bv | Resumo |
| CN102007209B (zh) | 2008-02-19 | 2013-11-13 | 阿姆斯特丹分子治疗(Amt)股份有限公司 | 细小病毒rep和cap蛋白在昆虫细胞中的表达优化 |
| EP2411505A4 (en) * | 2009-03-26 | 2013-01-30 | Univ California | MESENCHYMAL STEM CELLS PRODUCING INHIBITORY RNA WHICH CAN BE USED TO ACT IN THE COURSE OF A DISEASE |
| EP3421603B1 (en) * | 2009-05-02 | 2021-10-06 | Genzyme Corporation | Gene therapy for neurodegenerative disorders |
| WO2011122950A1 (en) | 2010-04-01 | 2011-10-06 | Amsterdam Molecular Therapeutics (Amt) Ip B.V. | Monomeric duplex aav vectors |
| EP3502254A1 (en) * | 2010-04-23 | 2019-06-26 | Cold Spring Harbor Laboratory | Novel structurally designed shrnas |
| WO2011158924A1 (ja) * | 2010-06-18 | 2011-12-22 | 独立行政法人国立精神・神経医療研究センター | 優性アレル発現抑制剤 |
| DK2744895T3 (en) | 2011-09-08 | 2016-01-04 | Uniqure Ip Bv | REMOVAL OF INFECTED VIRA FROM AAV PREPARATIONS |
| CN103224556B (zh) * | 2013-01-31 | 2015-04-15 | 清华大学 | 亨廷顿舞蹈症的靶位蛋白质及其编码基因和应用 |
| PT3237618T (pt) * | 2014-12-24 | 2019-07-04 | Uniqure Ip Bv | Supressão do gene huntingtina induzida por arni |
-
2015
- 2015-12-23 PT PT15817385T patent/PT3237618T/pt unknown
- 2015-12-23 RS RS20190744A patent/RS58862B1/sr unknown
- 2015-12-23 US US15/538,964 patent/US10174321B2/en active Active
- 2015-12-23 AU AU2015370903A patent/AU2015370903B2/en active Active
- 2015-12-23 EA EA201791417A patent/EA037696B1/ru unknown
- 2015-12-23 HU HUE15817385A patent/HUE043842T2/hu unknown
- 2015-12-23 EP EP15817385.6A patent/EP3237618B1/en active Active
- 2015-12-23 MX MX2017008500A patent/MX2017008500A/es unknown
- 2015-12-23 CN CN201580071228.2A patent/CN108064292B/zh active Active
- 2015-12-23 TR TR2019/08969T patent/TR201908969T4/tr unknown
- 2015-12-23 KR KR1020177020454A patent/KR102636633B1/ko active IP Right Grant
- 2015-12-23 ES ES15817385T patent/ES2732023T3/es active Active
- 2015-12-23 BR BR112017013573A patent/BR112017013573A2/pt active Search and Examination
- 2015-12-23 SI SI201530791T patent/SI3237618T1/sl unknown
- 2015-12-23 CA CA2971920A patent/CA2971920A1/en active Pending
- 2015-12-23 PL PL15817385T patent/PL3237618T3/pl unknown
- 2015-12-23 EP EP19167349.0A patent/EP3540063A1/en active Pending
- 2015-12-23 LT LTEP15817385.6T patent/LT3237618T/lt unknown
- 2015-12-23 DK DK15817385.6T patent/DK3237618T3/da active
- 2015-12-23 WO PCT/EP2015/081157 patent/WO2016102664A1/en active Application Filing
- 2015-12-23 JP JP2017552234A patent/JP6839094B2/ja active Active
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2017
- 2017-06-18 IL IL252990A patent/IL252990B/en unknown
- 2017-07-24 CO CONC2017/0007335A patent/CO2017007335A2/es unknown
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2018
- 2018-04-27 HK HK18105533.3A patent/HK1246344B/zh unknown
- 2018-11-16 US US16/193,908 patent/US10767180B2/en active Active
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2019
- 2019-05-31 HR HRP20190992TT patent/HRP20190992T1/hr unknown
- 2019-06-13 CY CY20191100616T patent/CY1121733T1/el unknown
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2020
- 2020-07-15 US US16/930,035 patent/US11371044B2/en active Active
- 2020-10-01 JP JP2020167128A patent/JP2021000136A/ja active Pending
-
2022
- 2022-05-24 US US17/751,934 patent/US20230119344A1/en active Pending
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