AU2019261438B2 - Expression of FOXP3 in edited CD34+ cells - Google Patents
Expression of FOXP3 in edited CD34+ cells Download PDFInfo
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- AU2019261438B2 AU2019261438B2 AU2019261438A AU2019261438A AU2019261438B2 AU 2019261438 B2 AU2019261438 B2 AU 2019261438B2 AU 2019261438 A AU2019261438 A AU 2019261438A AU 2019261438 A AU2019261438 A AU 2019261438A AU 2019261438 B2 AU2019261438 B2 AU 2019261438B2
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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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K35/14—Blood; Artificial blood
- A61K35/15—Cells of the myeloid line, e.g. granulocytes, basophils, eosinophils, neutrophils, leucocytes, monocytes, macrophages or mast cells; Myeloid precursor cells; Antigen-presenting cells, e.g. dendritic cells
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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
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- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
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- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4713—Autoimmune diseases, e.g. Insulin-dependent diabetes mellitus, multiple sclerosis, rheumathoid arthritis, systemic lupus erythematosus; Autoantigens
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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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- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0634—Cells from the blood or the immune system
- C12N5/0636—T lymphocytes
- C12N5/0637—Immunosuppressive T lymphocytes, e.g. regulatory T cells or Treg
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0634—Cells from the blood or the immune system
- C12N5/0645—Macrophages, e.g. Kuepfer cells in the liver; Monocytes
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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]
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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]
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2501/00—Active agents used in cell culture processes, e.g. differentation
- C12N2501/60—Transcription factors
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2510/00—Genetically modified cells
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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
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
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- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
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- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Immunology (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Hematology (AREA)
- Medicinal Chemistry (AREA)
- Cell Biology (AREA)
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- Gastroenterology & Hepatology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Toxicology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Developmental Biology & Embryology (AREA)
- Virology (AREA)
- Diabetes (AREA)
- Rehabilitation Therapy (AREA)
- Rheumatology (AREA)
- Mycology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| AU2024266824A AU2024266824A1 (en) | 2018-04-27 | 2024-11-22 | Expression of FOXP3 in edited CD34+ cells |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862663545P | 2018-04-27 | 2018-04-27 | |
| US62/663,545 | 2018-04-27 | ||
| PCT/US2019/029082 WO2019210042A1 (en) | 2018-04-27 | 2019-04-25 | Expression of foxp3 in edited cd34+ cells |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2024266824A Division AU2024266824A1 (en) | 2018-04-27 | 2024-11-22 | Expression of FOXP3 in edited CD34+ cells |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| AU2019261438A1 AU2019261438A1 (en) | 2020-09-10 |
| AU2019261438B2 true AU2019261438B2 (en) | 2024-08-22 |
Family
ID=68294731
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2019261438A Active AU2019261438B2 (en) | 2018-04-27 | 2019-04-25 | Expression of FOXP3 in edited CD34+ cells |
| AU2024266824A Pending AU2024266824A1 (en) | 2018-04-27 | 2024-11-22 | Expression of FOXP3 in edited CD34+ cells |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2024266824A Pending AU2024266824A1 (en) | 2018-04-27 | 2024-11-22 | Expression of FOXP3 in edited CD34+ cells |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US11713459B2 (enExample) |
| EP (1) | EP3784690A4 (enExample) |
| JP (2) | JP7575950B2 (enExample) |
| CN (1) | CN112218882A (enExample) |
| AU (2) | AU2019261438B2 (enExample) |
| CA (1) | CA3091688A1 (enExample) |
| IL (1) | IL277039A (enExample) |
| SG (1) | SG11202007878UA (enExample) |
| WO (1) | WO2019210042A1 (enExample) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2016427822B2 (en) | 2016-10-31 | 2025-01-23 | Seattle Children's Hospital (dba Seattle Children's Research Institute) | Method for treating autoimmune disease using CD4 T-cells with engineered stabilization of expression of endogennous FOXP3 gene |
| CN112218882A (zh) * | 2018-04-27 | 2021-01-12 | 西雅图儿童医院(Dba西雅图儿童研究所) | Foxp3在经编辑的cd34+细胞中的表达 |
| US20210253652A1 (en) * | 2018-04-27 | 2021-08-19 | Seattle Children's Hospital (dba Seattle Children's Research Institute) | Expression of human foxp3 in gene edited t cells |
| EP3912644A4 (en) * | 2019-01-18 | 2022-09-28 | Osaka University | THERAPEUTIC AGENT FOR DYSTROPHIC EPIDERMOLYSIS BULLOSA |
| CA3141159A1 (en) * | 2019-05-21 | 2020-11-26 | Sangamo Therapeutics, Inc. | Controlled transgene expression in regulatory t cells |
| NZ783217A (en) * | 2019-06-27 | 2025-08-29 | Seattle Children’S Hospital Dba Seattle Children’S Res Institute | Artificial antigen-specific immunoregulatory t (airt) cells |
| EP4055039A1 (en) | 2019-11-08 | 2022-09-14 | Sangamo Therapeutics, Inc. | Generation of engineered regulatory t cells |
| WO2021144692A1 (en) * | 2020-01-14 | 2021-07-22 | Crispr Therapeutics Ag | Methods for increased efficiency of homology-directed repair |
| WO2021163642A2 (en) * | 2020-02-13 | 2021-08-19 | The Board Of Trustees Of The Leland Stanford Junior University | Crispr-based foxp3 gene engineered t cells and hematopoietic stem cell precursors to treat ipex syndrome patients |
| AU2021368557A1 (en) * | 2020-10-27 | 2023-06-08 | Adoc Ssf, Llc | Compositions and methods for the treatment of cancer using next generation engineered t cell therapy |
| CN112851794B (zh) * | 2021-02-04 | 2023-05-23 | 苏州铂维生物科技有限公司 | 一种基于cd271的抗原表位及其应用 |
| GB202113674D0 (en) | 2021-09-24 | 2021-11-10 | Reflection Therapeutics Ltd | Targeted cell therapies |
| GB202113673D0 (en) | 2021-09-24 | 2021-11-10 | Reflection Therapeutics Ltd | Targeted cell therapies |
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| KR100996016B1 (ko) | 2000-09-20 | 2010-11-22 | 밀리포어 코포레이션 | 폴리뉴클레오티드 |
| JP4353701B2 (ja) | 2001-05-08 | 2009-10-28 | ダーウィン モレキュラー コーポレイション | Foxp3蛋白質を用いた霊長類における免疫機能の調節方法 |
| GB0614536D0 (en) | 2006-07-21 | 2006-08-30 | Metcalfe Susan M | Methods of controlling cellular response to LIF |
| EP2064350B1 (en) * | 2006-11-27 | 2013-01-02 | Ludwig Institute for Cancer Research Ltd. | Expression of foxp3 by cancer cells |
| US20100135974A1 (en) | 2007-01-31 | 2010-06-03 | Yeda Research And Development Co. Ltd. | Redirected, genetically-engineered t regulatory cells and their use in suppression of autoimmune and inflammatory disease |
| US9249423B2 (en) | 2007-02-02 | 2016-02-02 | Yale University | Method of de-differentiating and re-differentiating somatic cells using RNA |
| US20110123502A1 (en) * | 2007-02-21 | 2011-05-26 | Barry Simon C | Method for obtaining treg-cells |
| WO2008141282A2 (en) | 2007-05-11 | 2008-11-20 | The Regents Of The University Of Michigan | Materials and methods for foxp3 tumor suppression |
| WO2008154399A1 (en) | 2007-06-08 | 2008-12-18 | The Trustees Of The University Of Pennsylvania | Foxp3 oligomerization and intermolecular interactions |
| JP2011503232A (ja) | 2007-11-20 | 2011-01-27 | ザ ブリガム アンド ウィメンズ ホスピタル インコーポレイテッド | 免疫応答の調節 |
| JP2011513403A (ja) | 2008-03-03 | 2011-04-28 | コンバージ バイオテック, インコーポレイテッド | T細胞依存性免疫応答を調節する方法 |
| WO2012018930A1 (en) | 2010-08-03 | 2012-02-09 | University Of Miami | Methods of isolating and expanding human t regulatory cells and uses thereof for cellular therapy |
| WO2014180943A1 (en) | 2013-05-08 | 2014-11-13 | Vib Vzw | Mcl-1 as critical regulator of foxp3+ regulatory t cell survival, and use thereof to treat severe immune disorders |
| AU2014266833B2 (en) | 2013-05-13 | 2020-07-02 | Cellectis | Methods for engineering highly active T cell for immunotherapy |
| ES2883131T3 (es) * | 2013-05-29 | 2021-12-07 | Cellectis | Métodos para la modificación de células T para inmunoterapia utilizando el sistema de nucleasa CAS guiado por ARN |
| DK3004337T3 (da) | 2013-05-29 | 2017-11-13 | Cellectis | Fremgangsmåde til konstruktion af T-celler til immunoterapi ved brug af RNA-guidet Cas nuklease-system |
| JP6673838B2 (ja) | 2014-02-14 | 2020-04-01 | セレクティスCellectis | 免疫細胞と病的細胞の両方に存在する抗原を標的とするように操作された、免疫療法のための細胞 |
| US20170198306A1 (en) | 2014-03-21 | 2017-07-13 | Cellectis | Engineering mammalian genome using dna-guided argonaute interference systems (dais) |
| KR101835554B1 (ko) | 2014-06-24 | 2018-04-19 | 서울대학교 산학협력단 | C/ebp를 포함하는 유도성 t 조절세포 분화촉진 또는 안정화 조성물 및 방법 |
| EP3245294A4 (en) | 2015-01-12 | 2018-05-30 | Massachusetts Institute of Technology | Gene editing through microfluidic delivery |
| ES2880473T5 (es) * | 2015-01-30 | 2024-05-09 | Univ California | Suministro de proteínas en células hematopoyéticas primarias |
| HK1254190A1 (zh) | 2015-05-08 | 2019-07-12 | President And Fellows Of Harvard College | 通用供体干细胞和相关方法 |
| US11499168B2 (en) | 2016-04-25 | 2022-11-15 | Universitat Basel | Allele editing and applications thereof |
| WO2018031762A1 (en) | 2016-08-10 | 2018-02-15 | Duke University | Compositions, systems and methods for programming immune cell function through targeted gene regulation |
| CA3034094A1 (en) * | 2016-08-16 | 2018-02-22 | Bluebird Bio, Inc. | Il-10 receptor alpha homing endonuclease variants, compositions, and methods of use |
| WO2018073391A1 (en) * | 2016-10-19 | 2018-04-26 | Cellectis | Targeted gene insertion for improved immune cells therapy |
| WO2018081476A2 (en) | 2016-10-27 | 2018-05-03 | Intima Bioscience, Inc. | Viral methods of t cell therapy |
| AU2016427822B2 (en) | 2016-10-31 | 2025-01-23 | Seattle Children's Hospital (dba Seattle Children's Research Institute) | Method for treating autoimmune disease using CD4 T-cells with engineered stabilization of expression of endogennous FOXP3 gene |
| JP7206214B2 (ja) | 2016-12-13 | 2023-01-17 | シアトル チルドレンズ ホスピタル (ディービーエイ シアトル チルドレンズ リサーチ インスティテュート) | インビトロ及びインビボで操作された細胞において発現された化学誘導シグナル伝達複合体の外因性薬物活性化の方法 |
| CN109790518A (zh) | 2017-05-08 | 2019-05-21 | 中国科学院动物研究所 | 经修饰的t细胞、其制备方法及用途 |
| EP3672617A4 (en) * | 2017-08-22 | 2021-06-23 | The Regents of the University of California | LENTIVIRAL VECTORS EXPRESSING FOXP3 IN HEMATOPOIETIC STEM CELLS TO TREAT IMMUNITY DEFICIENCIES AND AUTOIMMUNE DISEASES |
| CN112218882A (zh) * | 2018-04-27 | 2021-01-12 | 西雅图儿童医院(Dba西雅图儿童研究所) | Foxp3在经编辑的cd34+细胞中的表达 |
| US20210253652A1 (en) | 2018-04-27 | 2021-08-19 | Seattle Children's Hospital (dba Seattle Children's Research Institute) | Expression of human foxp3 in gene edited t cells |
| WO2019241549A1 (en) | 2018-06-15 | 2019-12-19 | A2 Biotherapeutics, Inc. | Foxp3-expressing car-t regulatory cells |
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2019
- 2019-04-25 CN CN201980028370.7A patent/CN112218882A/zh active Pending
- 2019-04-25 EP EP19793428.4A patent/EP3784690A4/en active Pending
- 2019-04-25 AU AU2019261438A patent/AU2019261438B2/en active Active
- 2019-04-25 JP JP2020560400A patent/JP7575950B2/ja active Active
- 2019-04-25 SG SG11202007878UA patent/SG11202007878UA/en unknown
- 2019-04-25 US US16/981,223 patent/US11713459B2/en active Active
- 2019-04-25 WO PCT/US2019/029082 patent/WO2019210042A1/en not_active Ceased
- 2019-04-25 CA CA3091688A patent/CA3091688A1/en active Pending
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2020
- 2020-08-31 IL IL277039A patent/IL277039A/en unknown
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2023
- 2023-06-16 US US18/336,276 patent/US20240117352A1/en active Pending
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2024
- 2024-10-18 JP JP2024182581A patent/JP2025013884A/ja active Pending
- 2024-11-22 AU AU2024266824A patent/AU2024266824A1/en active Pending
Non-Patent Citations (2)
| Title |
|---|
| GOODWIN , M., et al., 2016, '123. Gene editing as a therapeutic approach to treat IPEX syndrome', Molecular Therapy, 24(1), pages S51-S52 * |
| KORNETE, M., et al., 2018, 'Highly efficient and versatile plasmid-based gene editing in primary T cells', Journal of Immunology, 200(7), pages 2489-2501 * |
Also Published As
| Publication number | Publication date |
|---|---|
| EP3784690A1 (en) | 2021-03-03 |
| EP3784690A4 (en) | 2022-01-19 |
| CA3091688A1 (en) | 2019-10-31 |
| SG11202007878UA (en) | 2020-09-29 |
| CN112218882A (zh) | 2021-01-12 |
| JP7575950B2 (ja) | 2024-10-31 |
| JP2025013884A (ja) | 2025-01-28 |
| AU2019261438A1 (en) | 2020-09-10 |
| AU2024266824A1 (en) | 2024-12-12 |
| JP2021521856A (ja) | 2021-08-30 |
| US20240117352A1 (en) | 2024-04-11 |
| IL277039A (en) | 2020-10-29 |
| US20210054376A1 (en) | 2021-02-25 |
| US11713459B2 (en) | 2023-08-01 |
| WO2019210042A1 (en) | 2019-10-31 |
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