JP2022514954A - キルスイッチを有するドナーt細胞 - Google Patents
キルスイッチを有するドナーt細胞 Download PDFInfo
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- JP2022514954A JP2022514954A JP2021536370A JP2021536370A JP2022514954A JP 2022514954 A JP2022514954 A JP 2022514954A JP 2021536370 A JP2021536370 A JP 2021536370A JP 2021536370 A JP2021536370 A JP 2021536370A JP 2022514954 A JP2022514954 A JP 2022514954A
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- Prior art keywords
- lymphocytes
- hprt
- nucleic acid
- cells
- acid sequence
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
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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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K40/00—Cellular immunotherapy
- A61K40/10—Cellular immunotherapy characterised by the cell type used
- A61K40/11—T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cells; Lymphokine-activated killer [LAK] cells
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K40/00—Cellular immunotherapy
- A61K40/20—Cellular immunotherapy characterised by the effect or the function of the cells
- A61K40/22—Immunosuppressive or immunotolerising
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K40/00—Cellular immunotherapy
- A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
- A61K40/41—Vertebrate antigens
- A61K40/418—Antigens related to induction of tolerance to non-self
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- 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
-
- 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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- C—CHEMISTRY; METALLURGY
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- 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
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing nucleic acids
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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
- C12N15/11—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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- 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
- C12N15/1137—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 against enzymes
-
- 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
- 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
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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
- C12N9/10—Transferases (2.)
- C12N9/1048—Glycosyltransferases (2.4)
- C12N9/1077—Pentosyltransferases (2.4.2)
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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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2121/00—Preparations for use in therapy
-
- 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
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
-
- 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
- C12N2500/00—Specific components of cell culture medium
- C12N2500/30—Organic components
-
- 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
- C12N2510/00—Genetically modified cells
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y204/00—Glycosyltransferases (2.4)
- C12Y204/02—Pentosyltransferases (2.4.2)
- C12Y204/02008—Hypoxanthine phosphoribosyltransferase (2.4.2.8)
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Medicinal Chemistry (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Immunology (AREA)
- Pharmacology & Pharmacy (AREA)
- Hematology (AREA)
- Cell Biology (AREA)
- Virology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Crystallography & Structural Chemistry (AREA)
- Diabetes (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Developmental Biology & Embryology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862784494P | 2018-12-23 | 2018-12-23 | |
| US62/784,494 | 2018-12-23 | ||
| PCT/US2019/068239 WO2020139800A1 (en) | 2018-12-23 | 2019-12-23 | Donor t-cells with kill switch |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022514954A true JP2022514954A (ja) | 2022-02-16 |
| JP2022514954A5 JP2022514954A5 (https=) | 2023-01-05 |
Family
ID=69326700
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021536370A Pending JP2022514954A (ja) | 2018-12-23 | 2019-12-23 | キルスイッチを有するドナーt細胞 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20210340563A1 (https=) |
| EP (1) | EP3897831A1 (https=) |
| JP (1) | JP2022514954A (https=) |
| KR (1) | KR20210118833A (https=) |
| CN (1) | CN113543848A (https=) |
| AU (1) | AU2019417700A1 (https=) |
| BR (1) | BR112021012318A2 (https=) |
| CA (1) | CA3123045A1 (https=) |
| WO (1) | WO2020139800A1 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2021263070A1 (en) * | 2020-06-26 | 2021-12-30 | Csl Behring Llc | Donor t-cells with kill switch |
| CN116162621B (zh) * | 2021-11-25 | 2025-09-16 | 中国科学院大连化学物理研究所 | Dna双链序列及应用和肺腺癌细胞增殖抑制剂或在肺腺癌药物 |
| CN115910357B (zh) * | 2022-11-14 | 2026-04-03 | 北京大学人民医院 | 预测HBsAg阳性患者造血干细胞移植后HBsAg清除的模型及装置 |
Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2017143266A1 (en) * | 2016-02-19 | 2017-08-24 | The Regents Of The University Of California | Short hairpin rna (shrna734) and use of same to positively select and eliminate genetically modified cells |
Family Cites Families (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5958928A (en) | 1995-03-27 | 1999-09-28 | Chugai Seiyaku Kabushiki Kaisha | Pharmaceutical agents containing methotrexate derivative |
| US6013516A (en) | 1995-10-06 | 2000-01-11 | The Salk Institute For Biological Studies | Vector and method of use for nucleic acid delivery to non-dividing cells |
| EP1191097A1 (en) | 2000-09-21 | 2002-03-27 | Leids Universitair Medisch Centrum | Induction of exon skipping in eukaryotic cells |
| EP2933332A1 (en) | 2004-06-28 | 2015-10-21 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| WO2007098089A2 (en) | 2006-02-17 | 2007-08-30 | Novacea, Inc. | Treatment of hyperproliferative diseases with methotrexate n-oxide and analogs |
| CN103849629B (zh) | 2006-06-21 | 2017-06-09 | 尤尼克尔Ip股份有限公司 | 具有经修饰的用于在昆虫细胞中产生aav的aav‑rep78翻译起始密码子的载体 |
| PL2291523T3 (pl) * | 2008-06-24 | 2015-05-29 | Csl Behring Gmbh | Czynnik VIII, czynnik von Willebranda lub ich kompleksy o wydłużonym okresie półtrwania in vivo |
| BR122020021379B1 (pt) | 2008-10-24 | 2021-05-11 | Sarepta Therapeutics, Inc. | oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular |
| TR201816523T4 (tr) | 2009-11-12 | 2018-11-21 | Univ Western Australia | Patolojilerin tedavisine yönelik antisens moleküller ve yöntemler. |
| US9283194B2 (en) | 2010-04-16 | 2016-03-15 | The Regents Of The University Of California | Methods for protease assisted protein delivery |
| DK2699247T3 (en) * | 2011-04-20 | 2018-06-14 | Univ California | PROCEDURE FOR COMBINED CONDITIONING AND CHEMOS SELECTION IN A SIMPLE CYCLE |
| US9993440B2 (en) | 2011-09-02 | 2018-06-12 | The Regents Of The University Of California | Enzyme responsive nanocapsules for protein delivery |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| CA2907095C (en) | 2012-03-16 | 2020-11-03 | The Regents Of The University Of California | A novel rnai molecule delivery platform based on single-sirna and shrna nanocapsules |
| HUE054260T2 (hu) | 2012-09-06 | 2021-08-30 | Univ Chicago | Antiszensz polinukleotidok exon-ugrás indukálására és eljárások disztrófiák kezelésére |
| JP6502261B2 (ja) | 2012-12-14 | 2019-04-17 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | ターゲティング遺伝子治療のためのウイルスベクターナノカプセル、およびその調製 |
| US20140329762A1 (en) | 2013-03-15 | 2014-11-06 | Sarepta Therapeutics, Inc. | Compositions for treating muscular dystrophy |
| CN105683376A (zh) * | 2013-05-15 | 2016-06-15 | 桑格摩生物科学股份有限公司 | 用于治疗遗传病状的方法和组合物 |
| WO2015075195A1 (en) * | 2013-11-22 | 2015-05-28 | Cellectis | Method of engineering chemotherapy drug resistant t-cells for immunotherapy |
| CN107530445B (zh) | 2014-11-26 | 2021-01-05 | 加利福尼亚大学董事会 | 用于蛋白质递送的隐形纳米胶囊 |
| CN107614012A (zh) * | 2015-04-24 | 2018-01-19 | 加利福尼亚大学董事会 | 使用工程化的细胞检测、监测或治疗疾病或病况的系统及制备和使用它们的方法 |
| HK1247963A1 (zh) | 2015-05-18 | 2018-10-05 | Csl贝林基因治疗股份有限公司 | 用於hiv的治疗的基因疗法及其用途 |
| EP3463428A4 (en) | 2016-05-24 | 2020-01-08 | The Regents of the University of California | GROWTH FACTOR NANO CAPSULES WITH TUNABLE RELEASE FOR BONE REGENERATION |
| CN111164211B (zh) * | 2017-07-18 | 2024-08-02 | 杰特贝林基因治疗股份有限公司 | 用于治疗β-血红蛋白病的组合物和方法 |
| JP7410856B2 (ja) * | 2017-07-18 | 2024-01-10 | シーエスエル・ベーリング・ジーン・セラピー・インコーポレイテッド | ドナー改変細胞の選択のための調節可能スイッチ |
-
2019
- 2019-12-23 KR KR1020217023088A patent/KR20210118833A/ko not_active Ceased
- 2019-12-23 JP JP2021536370A patent/JP2022514954A/ja active Pending
- 2019-12-23 AU AU2019417700A patent/AU2019417700A1/en not_active Abandoned
- 2019-12-23 WO PCT/US2019/068239 patent/WO2020139800A1/en not_active Ceased
- 2019-12-23 CA CA3123045A patent/CA3123045A1/en active Pending
- 2019-12-23 EP EP19842993.8A patent/EP3897831A1/en not_active Withdrawn
- 2019-12-23 CN CN201980092148.3A patent/CN113543848A/zh active Pending
- 2019-12-23 BR BR112021012318A patent/BR112021012318A2/pt not_active IP Right Cessation
-
2021
- 2021-06-21 US US17/353,572 patent/US20210340563A1/en not_active Abandoned
Patent Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2017143266A1 (en) * | 2016-02-19 | 2017-08-24 | The Regents Of The University Of California | Short hairpin rna (shrna734) and use of same to positively select and eliminate genetically modified cells |
Non-Patent Citations (2)
| Title |
|---|
| CHOUDHARY R. ET AL., PLOSONE, vol. Vol. 8:e59594, Table S1, JPN6023045398, 2013, ISSN: 0005390753 * |
| PORTER CC. ET AL., BLOOD, vol. 112, JPN6023045399, 2008, pages 4466 - 4474, ISSN: 0005390754 * |
Also Published As
| Publication number | Publication date |
|---|---|
| CA3123045A1 (en) | 2020-07-02 |
| US20210340563A1 (en) | 2021-11-04 |
| CN113543848A (zh) | 2021-10-22 |
| WO2020139800A1 (en) | 2020-07-02 |
| EP3897831A1 (en) | 2021-10-27 |
| AU2019417700A1 (en) | 2021-07-08 |
| KR20210118833A (ko) | 2021-10-01 |
| BR112021012318A2 (pt) | 2022-01-18 |
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