IL312266B2 - Polyploid adeno-associated virus vectors and methods of making and using the same - Google Patents
Polyploid adeno-associated virus vectors and methods of making and using the sameInfo
- Publication number
- IL312266B2 IL312266B2 IL312266A IL31226624A IL312266B2 IL 312266 B2 IL312266 B2 IL 312266B2 IL 312266 A IL312266 A IL 312266A IL 31226624 A IL31226624 A IL 31226624A IL 312266 B2 IL312266 B2 IL 312266B2
- Authority
- IL
- Israel
- Prior art keywords
- aav
- capsid
- nucleic acid
- virus
- vector
- Prior art date
Links
Classifications
-
- 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
- A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
-
- 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
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
-
- 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
- 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
-
- 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
- C12N2750/14123—Virus like particles [VLP]
-
- 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
- C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
-
- 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
- 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
- C12N2750/14151—Methods of production or purification of viral material
- C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Virology (AREA)
- Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Mycology (AREA)
- Epidemiology (AREA)
- Diabetes (AREA)
- Emergency Medicine (AREA)
- Endocrinology (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762471762P | 2017-03-15 | 2017-03-15 | |
| US201762520901P | 2017-06-16 | 2017-06-16 | |
| US201862630558P | 2018-02-14 | 2018-02-14 | |
| PCT/US2018/022725 WO2018170310A1 (en) | 2017-03-15 | 2018-03-15 | Polyploid adeno-associated virus vectors and methods of making and using the same |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| IL312266A IL312266A (en) | 2024-06-01 |
| IL312266B1 IL312266B1 (en) | 2024-11-01 |
| IL312266B2 true IL312266B2 (en) | 2025-03-01 |
Family
ID=63522576
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL268891A IL268891B2 (en) | 2017-03-15 | 2018-03-15 | Polyploid adeno-associated virus vectors and methods of making and using the same |
| IL312266A IL312266B2 (en) | 2017-03-15 | 2018-03-15 | Polyploid adeno-associated virus vectors and methods of making and using the same |
| IL316259A IL316259A (en) | 2017-03-15 | 2018-03-15 | Polyploid gland associated with virus vectors and methods for preparing and using the same. |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL268891A IL268891B2 (en) | 2017-03-15 | 2018-03-15 | Polyploid adeno-associated virus vectors and methods of making and using the same |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL316259A IL316259A (en) | 2017-03-15 | 2018-03-15 | Polyploid gland associated with virus vectors and methods for preparing and using the same. |
Country Status (8)
| Country | Link |
|---|---|
| EP (2) | EP3610023B1 (enExample) |
| JP (1) | JP7132934B2 (enExample) |
| CN (2) | CN110770346B (enExample) |
| AU (2) | AU2018234695B2 (enExample) |
| CA (1) | CA3054711A1 (enExample) |
| ES (1) | ES2966692T3 (enExample) |
| IL (3) | IL268891B2 (enExample) |
| WO (1) | WO2018170310A1 (enExample) |
Families Citing this family (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3151866B1 (en) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| PT3356390T (pt) | 2015-09-28 | 2021-04-21 | Univ Florida | Métodos e composições para vetores virais que se evadem a anticorpos |
| US12173305B2 (en) | 2016-05-26 | 2024-12-24 | University Of Iowa Research Foundation | cis and trans requirements for terminal resolution of human bocavirus 1 |
| US10550405B2 (en) | 2017-03-15 | 2020-02-04 | The University Of North Carolina At Chapel Hill | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
| JP2020530307A (ja) | 2017-06-30 | 2020-10-22 | インティマ・バイオサイエンス,インコーポレーテッド | 遺伝子治療のためのアデノ随伴ウイルスベクター |
| JP2021519581A (ja) | 2018-04-03 | 2021-08-12 | ストライドバイオ,インコーポレイテッド | 抗体を回避するウイルスベクター |
| SG11202009452WA (en) | 2018-04-03 | 2020-10-29 | Stridebio Inc | Antibody-evading virus vectors |
| JP7425738B2 (ja) | 2018-04-03 | 2024-01-31 | ギンコ バイオワークス インコーポレイテッド | 眼組織を標的とするウイルスベクター |
| NZ759802A (en) * | 2018-05-07 | 2025-08-29 | Univ North Carolina Chapel Hill | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
| JP2021523198A (ja) * | 2018-05-14 | 2021-09-02 | ビオマリン プハルマセウトイカル インコーポレイテッド | 幼若対象におけるaavベクターの安定的発現 |
| EP3880219A4 (en) * | 2018-11-16 | 2022-08-17 | Spark Therapeutics, Inc. | IN VITRO ASSAY FOR DETECTING ENHANCERS AND INHIBITORS OF ADENO-ASSOCIATED VIRUS (AAV) VECTOR TRANSDUCTION AND FOR DETECTING OR QUANTIFYING ANTI-AAV BINDING ANTIBODIES |
| CA3119771A1 (en) * | 2018-11-20 | 2020-05-28 | The Trustees Of The University Of Pennsylvania | Compositions and methods useful for targeting the blood-brain barrier |
| WO2020142236A1 (en) * | 2019-01-02 | 2020-07-09 | Intima Bioscience, Inc. | Modified adeno-associated viral vectors for use in genetic engineering |
| AU2020241888A1 (en) | 2019-03-21 | 2021-09-30 | Ginkgo Bioworks, Inc. | Recombinant adeno-associated virus vectors |
| CA3137078A1 (en) * | 2019-04-15 | 2020-10-22 | University Of Iowa Research Foundation | Methods and compositions for transgene expression |
| WO2020223236A1 (en) * | 2019-04-29 | 2020-11-05 | The Trustees Of The University Of Pennsylvania | Novel aav capsids and compositions containing same |
| KR20220083714A (ko) | 2019-10-17 | 2022-06-20 | 스트라이드바이오 인코포레이티드 | 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터 |
| CN111349148A (zh) * | 2020-03-13 | 2020-06-30 | 辉大(上海)生物科技有限公司 | 一种腺相关病毒载体及其用途 |
| EP4125977A4 (en) * | 2020-05-05 | 2024-09-25 | Duke University | CROSS-SPECIES COMPATIBLE ADENO-ASSOCIATED VIRUS COMPOSITIONS AND METHODS OF USE THEREOF |
| CN115927398B (zh) * | 2020-07-29 | 2024-11-19 | 北京三诺佳邑生物技术有限责任公司 | 一组肝靶向新型腺相关病毒的获得及其应用 |
| AR123288A1 (es) | 2020-08-19 | 2022-11-16 | Stridebio Inc | Vectores de virus adenoasociados para el tratamiento del síndrome de rett |
| CA3216491A1 (en) * | 2021-04-16 | 2022-10-20 | Asklepios Biopharmaceutical, Inc. | Rational polyploid aav virions that cross the blood brain barrier and elicit reduced humoral response |
| CA3242989A1 (en) * | 2021-12-21 | 2023-06-29 | Research Institute At Nationwide Children's Hospital | MATERIALS AND METHODS FOR THE TREATMENT OF MUSCULAR BELLY DYSTROPHY |
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| ES2026826A6 (es) | 1991-03-26 | 1992-05-01 | Ercros Sa | Procedimiento para la produccion de una vacuna subunidad contra el parvovirus canino y otros virus relacionados. |
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| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
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| CA2223837C (en) | 1995-06-07 | 2010-05-25 | University Of North Carolina At Chapel Hill | Aav transduction of myoblasts |
| US6040183A (en) | 1995-06-07 | 2000-03-21 | University Of North Carloina At Chapel Hill | Helper virus-free AAV production |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| US6083702A (en) | 1995-12-15 | 2000-07-04 | Intronn Holdings Llc | Methods and compositions for use in spliceosome mediated RNA trans-splicing |
| DE69636937T3 (de) | 1995-12-15 | 2011-01-05 | Virxsys Corp. | Durch trans-spaltung erhaltene therapeutische molekule |
| AU4645697A (en) | 1996-09-11 | 1998-04-02 | Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The | Aav4 vector and uses thereof |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| WO1999061601A2 (en) | 1998-05-28 | 1999-12-02 | The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Aav5 vector and uses thereof |
| EP1115880A2 (en) | 1998-09-22 | 2001-07-18 | University of Florida | Methods for large-scale production of recombinant aav vectors |
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| DE69941905D1 (de) | 1998-11-10 | 2010-02-25 | Univ North Carolina | Virusvektoren und verfahren für ihre herstellung und verabreichung. |
| US7314912B1 (en) | 1999-06-21 | 2008-01-01 | Medigene Aktiengesellschaft | AAv scleroprotein, production and use thereof |
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| AU2001268149B2 (en) | 2000-06-01 | 2005-08-18 | University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
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| ITRM20020253A1 (it) | 2002-05-08 | 2003-11-10 | Univ Roma | Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche. |
| FR2874384B1 (fr) | 2004-08-17 | 2010-07-30 | Genethon | Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables |
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| WO2007089632A2 (en) | 2006-01-27 | 2007-08-09 | The University Of North Carolina At Chapel Hill | Heparin and heparan sulfate binding chimeric vectors |
| EP2441770A1 (en) | 2006-02-10 | 2012-04-18 | The University of Cincinnati | Phosphatase inhibitor protein-1 as a regulator of cardiac function |
| CN101495624A (zh) * | 2006-04-28 | 2009-07-29 | 宾夕法尼亚州立大学托管会 | 衣壳免疫原性降低的经修饰aav载体及其用途 |
| CN101711164B (zh) | 2007-01-18 | 2014-06-04 | 密苏里-哥伦比亚大学 | 可修复肌纤维膜中的神经元型一氧化氮合酶的合成小/微-抗肌萎缩蛋白基因 |
| WO2009108274A2 (en) | 2008-02-26 | 2009-09-03 | The University Of North Carolina At Chapel Hill | Methods and compositions for adeno-associated virus (aav) with hi loop mutations |
| US20120093775A1 (en) * | 2009-03-27 | 2012-04-19 | Proyecto De Biomedicina Cima, S.L. | Methods and compositions for the treatment of cirrhosis and liver fibrosis |
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| ES2609860T3 (es) | 2011-10-28 | 2017-04-24 | The University Of North Carolina At Chapel Hill | Línea celular para la producción de virus adenoasociado |
| CA2870736C (en) | 2012-04-18 | 2021-11-02 | The Children's Hospital Of Philadelphia | Composition and methods for highly efficient gene transfer using aav capsid variants |
| US9163259B2 (en) * | 2012-05-04 | 2015-10-20 | Novartis Ag | Viral vectors for the treatment of retinal dystrophy |
| PH12016500162B1 (en) * | 2013-07-22 | 2024-02-21 | Childrens Hospital Philadelphia | Variant aav and compositions, methods and uses for gene trnsfer to cells, organs, and tissues |
| CA2990193A1 (en) * | 2015-06-23 | 2016-12-29 | The Children's Hospital Of Philadelphia | Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues |
-
2018
- 2018-03-15 CN CN201880031964.9A patent/CN110770346B/zh active Active
- 2018-03-15 EP EP18768025.1A patent/EP3610023B1/en active Active
- 2018-03-15 WO PCT/US2018/022725 patent/WO2018170310A1/en not_active Ceased
- 2018-03-15 IL IL268891A patent/IL268891B2/en unknown
- 2018-03-15 IL IL312266A patent/IL312266B2/en unknown
- 2018-03-15 JP JP2019545960A patent/JP7132934B2/ja active Active
- 2018-03-15 IL IL316259A patent/IL316259A/en unknown
- 2018-03-15 CA CA3054711A patent/CA3054711A1/en active Pending
- 2018-03-15 CN CN202311805114.9A patent/CN117801075A/zh active Pending
- 2018-03-15 AU AU2018234695A patent/AU2018234695B2/en active Active
- 2018-03-15 EP EP23195775.4A patent/EP4303225B1/en active Active
- 2018-03-15 ES ES18768025T patent/ES2966692T3/es active Active
-
2024
- 2024-10-14 AU AU2024227303A patent/AU2024227303A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP4303225B1 (en) | 2025-11-26 |
| CN110770346B (zh) | 2024-01-12 |
| IL268891B1 (en) | 2024-05-01 |
| EP4303225A3 (en) | 2024-04-10 |
| IL312266A (en) | 2024-06-01 |
| AU2024227303A1 (en) | 2024-10-31 |
| EP3610023B1 (en) | 2023-10-04 |
| JP2020511127A (ja) | 2020-04-16 |
| JP7132934B2 (ja) | 2022-09-07 |
| ES2966692T3 (es) | 2024-04-23 |
| CN117801075A (zh) | 2024-04-02 |
| CA3054711A1 (en) | 2018-09-20 |
| AU2018234695A1 (en) | 2019-09-12 |
| CN110770346A (zh) | 2020-02-07 |
| EP3610023A4 (en) | 2021-01-20 |
| IL312266B1 (en) | 2024-11-01 |
| AU2018234695B2 (en) | 2024-10-03 |
| IL268891A (en) | 2019-10-31 |
| EP3610023A1 (en) | 2020-02-19 |
| IL268891B2 (en) | 2024-09-01 |
| IL316259A (en) | 2024-12-01 |
| EP4303225A2 (en) | 2024-01-10 |
| WO2018170310A1 (en) | 2018-09-20 |
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