JP6811857B2 - B型肝炎ウイルスゲノムの認識配列に特異的な遺伝子操作メガヌクレアーゼ - Google Patents
B型肝炎ウイルスゲノムの認識配列に特異的な遺伝子操作メガヌクレアーゼ Download PDFInfo
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- JP6811857B2 JP6811857B2 JP2019520125A JP2019520125A JP6811857B2 JP 6811857 B2 JP6811857 B2 JP 6811857B2 JP 2019520125 A JP2019520125 A JP 2019520125A JP 2019520125 A JP2019520125 A JP 2019520125A JP 6811857 B2 JP6811857 B2 JP 6811857B2
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- meganuclease
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
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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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
- A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
- A61K9/51—Nanocapsules; Nanoparticles
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
- A61P31/20—Antivirals for DNA viruses
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
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- 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
- C12N15/52—Genes encoding for enzymes or proenzymes
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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
- C12N15/63—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
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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
- 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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- 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
- C12N2730/00—Reverse transcribing DNA viruses
- C12N2730/00011—Details
- C12N2730/10011—Hepadnaviridae
- C12N2730/10111—Orthohepadnavirus, e.g. hepatitis B virus
- C12N2730/10122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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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
- 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
-
- 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
- C12N2800/10—Plasmid DNA
- C12N2800/106—Plasmid DNA for vertebrates
- C12N2800/107—Plasmid DNA for vertebrates for mammalian
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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)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- General Engineering & Computer Science (AREA)
- Virology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Physics & Mathematics (AREA)
- Epidemiology (AREA)
- Oncology (AREA)
- Communicable Diseases (AREA)
- Gastroenterology & Hepatology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Nanotechnology (AREA)
- Optics & Photonics (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Enzymes And Modification Thereof (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2020207369A JP6919049B2 (ja) | 2016-10-14 | 2020-12-15 | B型肝炎ウイルスゲノムの認識配列に特異的な遺伝子操作メガヌクレアーゼ |
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662408356P | 2016-10-14 | 2016-10-14 | |
| US62/408,356 | 2016-10-14 | ||
| US201762452506P | 2017-01-31 | 2017-01-31 | |
| US62/452,506 | 2017-01-31 | ||
| US201762527196P | 2017-06-30 | 2017-06-30 | |
| US201762527159P | 2017-06-30 | 2017-06-30 | |
| US62/527,159 | 2017-06-30 | ||
| US62/527,196 | 2017-06-30 | ||
| PCT/US2017/056638 WO2018071849A2 (en) | 2016-10-14 | 2017-10-13 | Engineered meganucleases specific for recognition sequences in the hepatitis b virus genome |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020207369A Division JP6919049B2 (ja) | 2016-10-14 | 2020-12-15 | B型肝炎ウイルスゲノムの認識配列に特異的な遺伝子操作メガヌクレアーゼ |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019530462A JP2019530462A (ja) | 2019-10-24 |
| JP2019530462A5 JP2019530462A5 (enExample) | 2020-11-19 |
| JP6811857B2 true JP6811857B2 (ja) | 2021-01-13 |
Family
ID=60480360
Family Applications (5)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019520125A Active JP6811857B2 (ja) | 2016-10-14 | 2017-10-13 | B型肝炎ウイルスゲノムの認識配列に特異的な遺伝子操作メガヌクレアーゼ |
| JP2020207369A Active JP6919049B2 (ja) | 2016-10-14 | 2020-12-15 | B型肝炎ウイルスゲノムの認識配列に特異的な遺伝子操作メガヌクレアーゼ |
| JP2021120133A Pending JP2021180665A (ja) | 2016-10-14 | 2021-07-21 | B型肝炎ウイルスゲノムの認識配列に特異的な遺伝子操作メガヌクレアーゼ |
| JP2023115696A Pending JP2023153824A (ja) | 2016-10-14 | 2023-07-14 | B型肝炎ウイルスゲノムの認識配列に特異的な遺伝子操作メガヌクレアーゼ |
| JP2025087808A Pending JP2025138637A (ja) | 2016-10-14 | 2025-05-27 | B型肝炎ウイルスゲノムの認識配列に特異的な遺伝子操作メガヌクレアーゼ |
Family Applications After (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020207369A Active JP6919049B2 (ja) | 2016-10-14 | 2020-12-15 | B型肝炎ウイルスゲノムの認識配列に特異的な遺伝子操作メガヌクレアーゼ |
| JP2021120133A Pending JP2021180665A (ja) | 2016-10-14 | 2021-07-21 | B型肝炎ウイルスゲノムの認識配列に特異的な遺伝子操作メガヌクレアーゼ |
| JP2023115696A Pending JP2023153824A (ja) | 2016-10-14 | 2023-07-14 | B型肝炎ウイルスゲノムの認識配列に特異的な遺伝子操作メガヌクレアーゼ |
| JP2025087808A Pending JP2025138637A (ja) | 2016-10-14 | 2025-05-27 | B型肝炎ウイルスゲノムの認識配列に特異的な遺伝子操作メガヌクレアーゼ |
Country Status (21)
| Country | Link |
|---|---|
| US (6) | US10662416B2 (enExample) |
| EP (2) | EP4234691A3 (enExample) |
| JP (5) | JP6811857B2 (enExample) |
| KR (4) | KR20240123417A (enExample) |
| CN (2) | CN117402852A (enExample) |
| AU (2) | AU2017342536A1 (enExample) |
| BR (1) | BR112019007450A2 (enExample) |
| CA (1) | CA3040143A1 (enExample) |
| CL (1) | CL2019000993A1 (enExample) |
| CO (1) | CO2019003675A2 (enExample) |
| CR (1) | CR20190181A (enExample) |
| DK (1) | DK3526323T5 (enExample) |
| DO (1) | DOP2019000097A (enExample) |
| FI (1) | FI3526323T3 (enExample) |
| IL (3) | IL265921B2 (enExample) |
| MA (1) | MA46535A (enExample) |
| MX (2) | MX2019004349A (enExample) |
| PE (1) | PE20191353A1 (enExample) |
| PH (1) | PH12019500788A1 (enExample) |
| SG (2) | SG10201914029RA (enExample) |
| WO (1) | WO2018071849A2 (enExample) |
Cited By (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2021061841A (ja) * | 2016-10-14 | 2021-04-22 | プレシジョン バイオサイエンシズ,インク. | B型肝炎ウイルスゲノムの認識配列に特異的な遺伝子操作メガヌクレアーゼ |
| US11142750B2 (en) | 2018-04-12 | 2021-10-12 | Precision Biosciences, Inc. | Optimized engineered meganucleases having specificity for a recognition sequence in the Hepatitis B virus genome |
| US12410418B2 (en) | 2019-12-06 | 2025-09-09 | Precision Biosciences, Inc. | Optimized engineered meganucleases having specificity for a recognition sequence in the Hepatitis B virus genome |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN120843482A (zh) | 2019-05-07 | 2025-10-28 | 精密生物科学公司 | 用于识别序列的工程化大范围核酸酶的优化 |
| IL315295A (en) | 2019-09-30 | 2024-10-01 | Gilead Sciences Inc | Hbv vaccines and methods treating hbv |
| JP2023518433A (ja) | 2020-03-20 | 2023-05-01 | ギリアード サイエンシーズ, インコーポレイテッド | 4’-c-置換-2-ハロ-2’-デオキシアデノシンヌクレオシドのプロドラッグ並びにその製造法及び使用法 |
| US20230183664A1 (en) | 2020-05-11 | 2023-06-15 | Precision Biosciences, Inc. | Self-limiting viral vectors encoding nucleases |
| TWI815194B (zh) | 2020-10-22 | 2023-09-11 | 美商基利科學股份有限公司 | 介白素2-Fc融合蛋白及使用方法 |
| WO2022104062A1 (en) * | 2020-11-12 | 2022-05-19 | Precision Biosciences, Inc. | Engineered meganucleases having specificity for recognition sequences in the dystrophin gene |
| TW202310852A (zh) | 2021-05-13 | 2023-03-16 | 美商基利科學股份有限公司 | TLR8調節化合物及抗HBV siRNA療法之組合 |
| WO2024124044A1 (en) | 2022-12-07 | 2024-06-13 | The Brigham And Women’S Hospital, Inc. | Compositions and methods targeting sat1 for enhancing anti¬ tumor immunity during tumor progression |
| WO2024192141A1 (en) | 2023-03-13 | 2024-09-19 | Dana-Farber Cancer Institute, Inc. | Treatment of cancers having a drug-resistant mesenchymal cell state |
| WO2024226838A2 (en) | 2023-04-25 | 2024-10-31 | The Brigham And Women's Hospital, Inc. | Treatment of autoimmune diseases having a pathogenic t cell state |
| AU2024270764A1 (en) | 2023-05-15 | 2025-12-04 | Nchroma Bio, Inc. | Compositions and methods for epigenetic regulation of hbv gene expression |
| WO2025049788A1 (en) | 2023-08-29 | 2025-03-06 | The Broad Institute, Inc. | Optical genetic screens of intracellular and intercellular transcriptional circuits with perturb-fish |
| WO2025096916A1 (en) | 2023-11-03 | 2025-05-08 | The Broad Institute, Inc. | Multi-site editing in living cells |
| WO2025129158A1 (en) | 2023-12-15 | 2025-06-19 | The Broad Institute, Inc. | Engineered arc delivery vesicles and uses thereof |
| US20250345390A1 (en) | 2024-05-13 | 2025-11-13 | Gilead Sciences, Inc. | Combination therapies |
| WO2025240242A1 (en) | 2024-05-13 | 2025-11-20 | Gilead Sciences, Inc. | Combination therapies with ribavirin |
| US20250345389A1 (en) | 2024-05-13 | 2025-11-13 | Gilead Sciences, Inc. | Combination therapies |
| WO2025240246A1 (en) | 2024-05-13 | 2025-11-20 | Gilead Sciences, Inc. | Combination therapies with ribavirin |
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| JP2021061841A (ja) * | 2016-10-14 | 2021-04-22 | プレシジョン バイオサイエンシズ,インク. | B型肝炎ウイルスゲノムの認識配列に特異的な遺伝子操作メガヌクレアーゼ |
| US11274285B2 (en) | 2016-10-14 | 2022-03-15 | Precision Biosciences, Inc. | Engineered meganucleases specific for recognition sequences in the Hepatitis B virus genome |
| US11142750B2 (en) | 2018-04-12 | 2021-10-12 | Precision Biosciences, Inc. | Optimized engineered meganucleases having specificity for a recognition sequence in the Hepatitis B virus genome |
| US11788077B2 (en) | 2018-04-12 | 2023-10-17 | Precision Biosciences, Inc. | Polynucleotides encoding optimized engineered meganucleases having specificity for a recognition sequence in the Hepatitis B virus genome |
| US12410418B2 (en) | 2019-12-06 | 2025-09-09 | Precision Biosciences, Inc. | Optimized engineered meganucleases having specificity for a recognition sequence in the Hepatitis B virus genome |
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