JP2000506726A - 組換えａａｖベクターに基づく形質導入系およびその使用 - Google Patents
組換えａａｖベクターに基づく形質導入系およびその使用Info
- Publication number
- JP2000506726A JP2000506726A JP9531353A JP53135397A JP2000506726A JP 2000506726 A JP2000506726 A JP 2000506726A JP 9531353 A JP9531353 A JP 9531353A JP 53135397 A JP53135397 A JP 53135397A JP 2000506726 A JP2000506726 A JP 2000506726A
- Authority
- JP
- Japan
- Prior art keywords
- rep
- transduction system
- cells
- dna
- aav
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Ceased
Links
- 238000010361 transduction Methods 0.000 title claims abstract description 42
- 230000026683 transduction Effects 0.000 title claims abstract description 42
- 239000013607 AAV vector Substances 0.000 title claims abstract description 26
- 108090000623 proteins and genes Proteins 0.000 claims abstract description 37
- 102000004169 proteins and genes Human genes 0.000 claims abstract description 27
- 210000004027 cell Anatomy 0.000 claims description 52
- 108020004414 DNA Proteins 0.000 claims description 42
- 239000000126 substance Substances 0.000 claims description 25
- 239000002245 particle Substances 0.000 claims description 12
- 101150044789 Cap gene Proteins 0.000 claims description 7
- 230000001939 inductive effect Effects 0.000 claims description 7
- 238000001415 gene therapy Methods 0.000 claims description 4
- 238000000034 method Methods 0.000 claims description 4
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims description 3
- 210000004881 tumor cell Anatomy 0.000 claims description 3
- 230000002950 deficient Effects 0.000 claims description 2
- 108091029865 Exogenous DNA Proteins 0.000 claims 2
- 239000003153 chemical reaction reagent Substances 0.000 abstract 1
- 241000702421 Dependoparvovirus Species 0.000 description 20
- 241000700605 Viruses Species 0.000 description 9
- 101150066583 rep gene Proteins 0.000 description 8
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- 108010039918 Polylysine Proteins 0.000 description 4
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- 101100524321 Adeno-associated virus 2 (isolate Srivastava/1982) Rep68 gene Proteins 0.000 description 3
- 101100524324 Adeno-associated virus 2 (isolate Srivastava/1982) Rep78 gene Proteins 0.000 description 3
- 239000004098 Tetracycline Substances 0.000 description 3
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- 101100524317 Adeno-associated virus 2 (isolate Srivastava/1982) Rep40 gene Proteins 0.000 description 2
- 101100524319 Adeno-associated virus 2 (isolate Srivastava/1982) Rep52 gene Proteins 0.000 description 2
- 108010068250 Herpes Simplex Virus Protein Vmw65 Proteins 0.000 description 2
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- 230000002463 transducing effect Effects 0.000 description 2
- 208000024719 uterine cervix neoplasm Diseases 0.000 description 2
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- 102000004506 Blood Proteins Human genes 0.000 description 1
- 108010017384 Blood Proteins Proteins 0.000 description 1
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- 102100023321 Ceruloplasmin Human genes 0.000 description 1
- 201000003883 Cystic fibrosis Diseases 0.000 description 1
- 102000053602 DNA Human genes 0.000 description 1
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- 108010054147 Hemoglobins Proteins 0.000 description 1
- 208000031220 Hemophilia Diseases 0.000 description 1
- 208000009292 Hemophilia A Diseases 0.000 description 1
- 102000014150 Interferons Human genes 0.000 description 1
- 108010050904 Interferons Proteins 0.000 description 1
- 102000015696 Interleukins Human genes 0.000 description 1
- 108010063738 Interleukins Proteins 0.000 description 1
- 208000024556 Mendelian disease Diseases 0.000 description 1
- 229930193140 Neomycin Natural products 0.000 description 1
- 108700026244 Open Reading Frames Proteins 0.000 description 1
- 208000018737 Parkinson disease Diseases 0.000 description 1
- 241000701945 Parvoviridae Species 0.000 description 1
- 108020004682 Single-Stranded DNA Proteins 0.000 description 1
- 108060008682 Tumor Necrosis Factor Proteins 0.000 description 1
- 230000005856 abnormality Effects 0.000 description 1
- 150000001413 amino acids Chemical class 0.000 description 1
- 239000003242 anti bacterial agent Substances 0.000 description 1
- 230000003115 biocidal effect Effects 0.000 description 1
- 201000011510 cancer Diseases 0.000 description 1
- 238000003776 cleavage reaction Methods 0.000 description 1
- 238000012217 deletion Methods 0.000 description 1
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- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 1
- 238000004520 electroporation Methods 0.000 description 1
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- 230000006698 induction Effects 0.000 description 1
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- 229960004927 neomycin Drugs 0.000 description 1
- 239000002773 nucleotide Substances 0.000 description 1
- 125000003729 nucleotide group Chemical group 0.000 description 1
- 210000000056 organ Anatomy 0.000 description 1
- 229920002714 polyornithine Polymers 0.000 description 1
- 108010055896 polyornithine Proteins 0.000 description 1
- 230000023603 positive regulation of transcription initiation, DNA-dependent Effects 0.000 description 1
- 108020003175 receptors Proteins 0.000 description 1
- 102220201851 rs143406017 Human genes 0.000 description 1
- 102220240796 rs553605556 Human genes 0.000 description 1
- 230000007017 scission Effects 0.000 description 1
- 241000894007 species Species 0.000 description 1
- 101150061166 tetR gene Proteins 0.000 description 1
- 230000009466 transformation Effects 0.000 description 1
- 102000003390 tumor necrosis factor Human genes 0.000 description 1
- 241000701161 unidentified adenovirus Species 0.000 description 1
- 241001529453 unidentified herpesvirus Species 0.000 description 1
- 239000013598 vector Substances 0.000 description 1
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
- C12N15/63—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
-
- 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
- A61P25/16—Anti-Parkinson drugs
-
- 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
-
- 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
- A61P31/14—Antivirals for RNA viruses
- A61P31/18—Antivirals for RNA viruses for HIV
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
- A61P37/04—Immunostimulants
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Animal Behavior & Ethology (AREA)
- Genetics & Genomics (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Immunology (AREA)
- Virology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Neurosurgery (AREA)
- Physics & Mathematics (AREA)
- Neurology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Oncology (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Communicable Diseases (AREA)
- Tropical Medicine & Parasitology (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- AIDS & HIV (AREA)
- Psychology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| DE19608753A DE19608753C1 (de) | 1996-03-06 | 1996-03-06 | Transduktionssystem und seine Verwendung |
| DE19608753.8 | 1996-03-06 | ||
| PCT/DE1997/000447 WO1997032989A2 (de) | 1996-03-06 | 1997-03-06 | Transduktionssystem beruhend auf rekombinantem aav-vektor und seine verwendung |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2000506726A true JP2000506726A (ja) | 2000-06-06 |
| JP2000506726A5 JP2000506726A5 (cg-RX-API-DMAC7.html) | 2004-11-25 |
Family
ID=7787450
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP9531353A Ceased JP2000506726A (ja) | 1996-03-06 | 1997-03-06 | 組換えａａｖベクターに基づく形質導入系およびその使用 |
Country Status (5)
| Country | Link |
|---|---|
| US (3) | US6207453B1 (cg-RX-API-DMAC7.html) |
| EP (1) | EP0891429A2 (cg-RX-API-DMAC7.html) |
| JP (1) | JP2000506726A (cg-RX-API-DMAC7.html) |
| DE (1) | DE19608753C1 (cg-RX-API-DMAC7.html) |
| WO (1) | WO1997032989A2 (cg-RX-API-DMAC7.html) |
Families Citing this family (196)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DE19608753C1 (de) * | 1996-03-06 | 1997-06-26 | Medigene Gmbh | Transduktionssystem und seine Verwendung |
| JP2006524991A (ja) * | 2003-05-08 | 2006-11-09 | エクサイト セラピーズ インコーポレーティッド | 抗原特異的ｔ細胞の作製および単離の方法 |
| WO2005044932A1 (en) * | 2003-11-11 | 2005-05-19 | Canon Kabushiki Kaisha | An ink comprising a block copolymer dispersing agent having a hydrophilic and a hydrophobic segment and an ink-applying process and apparatus using the same |
| AU2005287393A1 (en) * | 2004-06-18 | 2006-03-30 | The University Of Montana | AAV mediated gene delivery to cochlear cells |
| US20100080784A1 (en) * | 2008-09-12 | 2010-04-01 | Torrey Pines Institute For Molecular Studies | Methods for treating cachexia and lymphopenia |
| WO2013163628A2 (en) | 2012-04-27 | 2013-10-31 | Duke University | Genetic correction of mutated genes |
| US9828582B2 (en) | 2013-03-19 | 2017-11-28 | Duke University | Compositions and methods for the induction and tuning of gene expression |
| RU2763795C2 (ru) | 2014-04-23 | 2022-01-11 | Джуно Терапьютикс, Инк. | Способы выделения, культивирования и генетической инженерии популяций клеток иммунной системы для адоптивной терапии |
| AU2015289644A1 (en) | 2014-07-15 | 2017-02-02 | Juno Therapeutics, Inc. | Engineered cells for adoptive cell therapy |
| TWI751102B (zh) | 2014-08-28 | 2022-01-01 | 美商奇諾治療有限公司 | 對ｃｄ１９具專一性之抗體及嵌合抗原受體 |
| IL293714A (en) | 2014-10-20 | 2022-08-01 | Juno Therapeutics Inc | Methods and compositions for dosing in adoptive cell therapy |
| SG10202002324SA (en) | 2014-11-05 | 2020-05-28 | Juno Therapeutics Inc | Methods for transduction and cell processing |
| WO2016090190A1 (en) | 2014-12-03 | 2016-06-09 | Juno Therapeutics, Inc. | Methods and compositions for adoptive cell therapy |
| US20160208018A1 (en) | 2015-01-16 | 2016-07-21 | Juno Therapeutics, Inc. | Antibodies and chimeric antigen receptors specific for ror1 |
| WO2016130600A2 (en) | 2015-02-09 | 2016-08-18 | Duke University | Compositions and methods for epigenome editing |
| WO2016166568A1 (en) | 2015-04-16 | 2016-10-20 | Juno Therapeutics Gmbh | Methods, kits and apparatus for expanding a population of cells |
| CN108174607A (zh) | 2015-05-29 | 2018-06-15 | 朱诺治疗学股份有限公司 | 用于遗传工程改造的细胞中调节抑制性相互作用的组合物和方法 |
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| EP4644567A2 (en) | 2015-11-30 | 2025-11-05 | Duke University | Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use |
| ES2928167T3 (es) | 2015-12-03 | 2022-11-15 | Juno Therapeutics Inc | Composiciones y métodos para reducir las respuestas inmunitarias contra receptores de antígenos quiméricos |
| EP3383892B1 (en) | 2015-12-03 | 2022-12-21 | Juno Therapeutics, Inc. | Modified chimeric receptors and related compositions and methods |
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| EP3430549A1 (en) | 2016-03-16 | 2019-01-23 | Juno Therapeutics, Inc. | Methods for adaptive design of a treatment regimen and related treatments |
| MA43758A (fr) | 2016-03-16 | 2018-11-28 | Yuan Ji | Procédés pour déterminer le dosage d'un agent thérapeutique et traitements associés |
| MX2018011480A (es) | 2016-03-22 | 2019-03-28 | Seattle Children´S Hospital Dba Seattle Children´S Res Institute | Metodos de intervencion temprana para prevenir o aminorar toxicidad. |
| US20190127713A1 (en) | 2016-04-13 | 2019-05-02 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
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| WO2018017754A1 (en) | 2016-07-19 | 2018-01-25 | Duke University | Therapeutic applications of cpf1-based genome editing |
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Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5436146A (en) * | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
| WO1991018088A1 (en) * | 1990-05-23 | 1991-11-28 | The United States Of America, Represented By The Secretary, United States Department Of Commerce | Adeno-associated virus (aav)-based eucaryotic vectors |
| US5587308A (en) * | 1992-06-02 | 1996-12-24 | The United States Of America As Represented By The Department Of Health & Human Services | Modified adeno-associated virus vector capable of expression from a novel promoter |
| US5478745A (en) * | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| NZ263550A (en) * | 1993-03-19 | 1996-12-20 | Boehringer Ingelheim Int | Preparing cancer vaccines containing autologous tumour cells characterised in that tumour cells or firbroblasts are cultivated and transfected ex vivo with a composition of dna and a dna binding molecule conjugate |
| US5693531A (en) * | 1993-11-24 | 1997-12-02 | The United States Of America As Represented By The Department Of Health And Human Services | Vector systems for the generation of adeno-associated virus particles |
| FR2716893B1 (fr) * | 1994-03-03 | 1996-04-12 | Rhone Poulenc Rorer Sa | Virus recombinants, leur préparation et leur utilisation thérapeutique. |
| US6342390B1 (en) * | 1994-11-23 | 2002-01-29 | The United States Of America As Represented By The Secretary Of Health And Human Services | Lipid vesicles containing adeno-associated virus rep protein for transgene integration and gene therapy |
| DE19608753C1 (de) * | 1996-03-06 | 1997-06-26 | Medigene Gmbh | Transduktionssystem und seine Verwendung |
-
1996
- 1996-03-06 DE DE19608753A patent/DE19608753C1/de not_active Expired - Fee Related
-
1997
- 1997-03-06 US US09/142,289 patent/US6207453B1/en not_active Expired - Fee Related
- 1997-03-06 WO PCT/DE1997/000447 patent/WO1997032989A2/de not_active Ceased
- 1997-03-06 EP EP97918010A patent/EP0891429A2/de not_active Withdrawn
- 1997-03-06 JP JP9531353A patent/JP2000506726A/ja not_active Ceased
-
2001
- 2001-01-31 US US09/773,302 patent/US6440742B1/en not_active Expired - Fee Related
-
2002
- 2002-08-12 US US10/218,280 patent/US20020192826A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| WO1997032989A3 (de) | 1998-01-15 |
| DE19608753C1 (de) | 1997-06-26 |
| WO1997032989A2 (de) | 1997-09-12 |
| US6440742B1 (en) | 2002-08-27 |
| US20020028514A1 (en) | 2002-03-07 |
| EP0891429A2 (de) | 1999-01-20 |
| US6207453B1 (en) | 2001-03-27 |
| US20020192826A1 (en) | 2002-12-19 |
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