DE60333487D1 - Therapeutische retrovirus-vektoren für gentherapie - Google Patents
Therapeutische retrovirus-vektoren für gentherapieInfo
- Publication number
- DE60333487D1 DE60333487D1 DE60333487T DE60333487T DE60333487D1 DE 60333487 D1 DE60333487 D1 DE 60333487D1 DE 60333487 T DE60333487 T DE 60333487T DE 60333487 T DE60333487 T DE 60333487T DE 60333487 D1 DE60333487 D1 DE 60333487D1
- Authority
- DE
- Germany
- Prior art keywords
- gene therapy
- hematopoietic
- retrovirus vectors
- cells
- methods
- 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.)
- Expired - Lifetime
Links
- 238000001415 gene therapy Methods 0.000 title abstract 4
- 239000013598 vector Substances 0.000 title abstract 2
- 230000001225 therapeutic effect Effects 0.000 title 1
- 241001430294 unidentified retrovirus Species 0.000 title 1
- 230000000925 erythroid effect Effects 0.000 abstract 2
- 230000003394 haemopoietic effect Effects 0.000 abstract 2
- 238000000034 method Methods 0.000 abstract 2
- 210000004027 cell Anatomy 0.000 abstract 1
- 210000001671 embryonic stem cell Anatomy 0.000 abstract 1
- 210000003743 erythrocyte Anatomy 0.000 abstract 1
- 210000003958 hematopoietic stem cell Anatomy 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 239000002243 precursor Substances 0.000 abstract 1
- 230000001177 retroviral effect Effects 0.000 abstract 1
Classifications
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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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K67/00—Rearing or breeding animals, not otherwise provided for; New breeds of animals
- A01K67/027—New breeds of vertebrates
- A01K67/0275—Genetically modified vertebrates, e.g. transgenic
- A01K67/0278—Humanized animals, e.g. knockin
-
- 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
- A61K38/41—Porphyrin- or corrin-ring-containing peptides
- A61K38/42—Haemoglobins; Myoglobins
-
- 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
-
- 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
- A61P7/06—Antianaemics
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/795—Porphyrin- or corrin-ring-containing peptides
- C07K14/805—Haemoglobins; Myoglobins
-
- 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
- C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
-
- 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
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2207/00—Modified animals
- A01K2207/15—Humanized animals
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
-
- 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
-
- 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
- C12N15/867—Retroviral 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
- C12N2510/00—Genetically modified cells
-
- 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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
- C12N2740/15041—Use of virus, viral particle or viral elements as a vector
- C12N2740/15043—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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/16011—Human Immunodeficiency Virus, HIV
-
- 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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/16011—Human Immunodeficiency Virus, HIV
- C12N2740/16041—Use of virus, viral particle or viral elements as a vector
- C12N2740/16043—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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/40—Vector systems having a special element relevant for transcription being an insulator
Applications Claiming Priority (4)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US43332102P | 2002-12-13 | 2002-12-13 | |
US47582203P | 2003-06-04 | 2003-06-04 | |
US51331203P | 2003-10-21 | 2003-10-21 | |
PCT/US2003/039526 WO2004054512A2 (en) | 2002-12-13 | 2003-12-11 | Therapeutic retroviral vectors for gene therapy |
Publications (1)
Publication Number | Publication Date |
---|---|
DE60333487D1 true DE60333487D1 (de) | 2010-09-02 |
Family
ID=32600914
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
DE60333487T Expired - Lifetime DE60333487D1 (de) | 2002-12-13 | 2003-12-11 | Therapeutische retrovirus-vektoren für gentherapie |
Country Status (9)
Country | Link |
---|---|
US (6) | US7901671B2 (de) |
EP (1) | EP1581056B1 (de) |
AT (1) | ATE474460T1 (de) |
AU (1) | AU2003296958A1 (de) |
CY (1) | CY1112327T1 (de) |
DE (1) | DE60333487D1 (de) |
ES (1) | ES2348868T3 (de) |
HK (1) | HK1082379A1 (de) |
WO (1) | WO2004054512A2 (de) |
Families Citing this family (68)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP1581056B1 (de) * | 2002-12-13 | 2010-07-21 | Genetix Pharmaceuticals Inc. | Therapeutische retrovirus-vektoren für gentherapie |
WO2007109583A2 (en) | 2006-03-17 | 2007-09-27 | The Government Of The United States Of America As Represented By The Secretary, Department Of Health And Human Services | Compositions and methods for prevention or treatment of neoplastic disease in a mammalian subject |
US20090144270A1 (en) * | 2007-11-29 | 2009-06-04 | Jack Thacher Leonard | Methods for the prevention of diseases |
GB0812191D0 (en) * | 2008-07-03 | 2008-08-13 | Greater Glasgow Health Board | Modified cells and methods of monitoring their viability |
ES2631458T3 (es) | 2010-03-04 | 2017-08-31 | Interna Technologies B.V. | Molécula de ARNmi definida por su fuente y sus usos terapéuticos en el cáncer asociado a la EMT |
CA2804599C (en) | 2010-07-06 | 2023-01-31 | Interna Technologies Bv | Mirna and its diagnostic and therapeutic uses in diseases or conditions associated with melanoma, or in diseases or conditions associated with activated braf pathway |
EP2474617A1 (de) | 2011-01-11 | 2012-07-11 | InteRNA Technologies BV | MIR zur Behandlung von Neoangiogenese |
US9789139B2 (en) | 2011-06-10 | 2017-10-17 | Bluebird Bio, Inc. | Gene therapy vectors for adrenoleukodystrophy and adrenomyeloneuropathy |
AU2011377617B2 (en) | 2011-09-23 | 2018-03-08 | Bluebird Bio, Inc. | Improved gene therapy methods |
KR102011532B1 (ko) | 2011-09-30 | 2019-08-16 | 블루버드 바이오, 인코포레이티드. | 개선된 바이러스 형질도입을 위한 화합물 |
US10174314B2 (en) | 2011-12-22 | 2019-01-08 | Interna Technologies B.V. | MiRNA for treating head and neck cancer |
EP2825048A4 (de) * | 2012-03-13 | 2015-10-14 | Anthrogenesis Corp | Modifizierte erythrozytenvorläuferzellen und verwendungen davon |
WO2014043131A1 (en) * | 2012-09-14 | 2014-03-20 | The Regents Of The University Of California | Lentiviral vector for stem cell gene therapy of sickle cell disease |
EP2917348A1 (de) | 2012-11-06 | 2015-09-16 | InteRNA Technologies B.V. | Kombination zur behandlung von erkrankungen oder zuständen im zusammenhang mit melanom oder behandlung von erkrankungen und zuständen im zusammenhang mit aktivierten b-raf-signalweg |
SG11201504038XA (en) | 2012-11-27 | 2015-06-29 | Childrens Medical Center | Targeting bcl11a distal regulatory elements for fetal hemoglobin reinduction |
WO2015073587A2 (en) | 2013-11-18 | 2015-05-21 | Rubius Therapeutics, Inc. | Synthetic membrane-receiver complexes |
EP3125927B1 (de) | 2014-04-01 | 2021-01-27 | Rubius Therapeutics, Inc. | Verfahren und zusammensetzungen zur immunmodulation |
JP6514717B2 (ja) | 2014-04-25 | 2019-05-15 | ザ チルドレンズ メディカル センター コーポレーション | 異常ヘモグロビン症を治療するための組成物および方法 |
FI3186395T4 (fi) * | 2014-08-26 | 2023-05-25 | Kantasolujen siirto kantasoluihin kohdistuvan aineen ja immunoregulatorisen signaloinnin modulaation yhdistelmällä | |
WO2016037163A1 (en) | 2014-09-07 | 2016-03-10 | Selecta Biosciences, Inc. | Methods and compositions for attenuating gene therapy anti-viral transfer vector immune responses |
EP3191596B1 (de) | 2014-09-11 | 2019-10-09 | The U.S.A. as represented by the Secretary, Department of Health and Human Services | Lentiviraler vektor zur behandlung von hämoglobinerkrankungen |
AU2016209313B2 (en) * | 2015-01-21 | 2021-10-14 | Cornell University | Viral vectors for prophylaxis and therapy of hemoglobinopathies |
ES2835861T3 (es) | 2015-05-08 | 2021-06-23 | Childrens Medical Ct Corp | Direccionamiento de regiones funcionales del potenciador de BCL11A para la reinducción de hemoglobina fetal |
GB201600328D0 (en) | 2016-01-08 | 2016-02-24 | Univ Oslo Hf | Anti-CD37 chimeric antigen receptors and immune cells expressing them |
US11326183B2 (en) | 2016-02-12 | 2022-05-10 | Bluebird Bio, Inc. | VCN enhancer compositions and methods of using the same |
EP4151720A1 (de) | 2016-02-12 | 2023-03-22 | Bluebird Bio, Inc. | Vcn-förderer-zusammensetzungen und verfahren zur verwendung davon |
US11446398B2 (en) | 2016-04-11 | 2022-09-20 | Obsidian Therapeutics, Inc. | Regulated biocircuit systems |
CA3043768A1 (en) | 2016-11-29 | 2018-06-07 | PureTech Health LLC | Exosomes for delivery of therapeutic agents |
AU2018205496A1 (en) | 2017-01-07 | 2019-07-25 | Selecta Biosciences, Inc. | Patterned dosing of immunosuppressants coupled to synthetic nanocarriers |
EP3574104A1 (de) | 2017-01-30 | 2019-12-04 | The U.S.A. As Represented By The Secretary, Department Of Health And Human Services | Rekombinante virusvektoren zur behandlung der glykogenspeicherkrankheit |
TW201839136A (zh) | 2017-02-06 | 2018-11-01 | 瑞士商諾華公司 | 治療血色素異常症之組合物及方法 |
US11261441B2 (en) | 2017-03-29 | 2022-03-01 | Bluebird Bio, Inc. | Vectors and compositions for treating hemoglobinopathies |
WO2018218135A1 (en) | 2017-05-25 | 2018-11-29 | The Children's Medical Center Corporation | Bcl11a guide delivery |
WO2019075360A1 (en) | 2017-10-13 | 2019-04-18 | Selecta Biosciences, Inc. | METHODS AND COMPOSITIONS FOR MITIGATING ANTI-VECTOR VIRAL TRANSFER IGM RESPONSES |
US11497762B2 (en) | 2017-11-03 | 2022-11-15 | Interna Technologies B.V. | MiRNA molecule, equivalent, antagomir, or source thereof for treating and/or diagnosing a condition and/or a disease associated with neuronal deficiency or for neuronal (re)generation |
KR20200139679A (ko) | 2018-02-26 | 2020-12-14 | 안톨알엑스, 인크. | 면역관용성 리포솜 및 그의 사용 방법 |
US11344008B1 (en) * | 2018-03-11 | 2022-05-31 | Hai Xing Chen | Production of human DNaseI in erythrocytes of transgenic non-human mammal using erythroid-specific promoter |
EP3806888B1 (de) | 2018-06-12 | 2024-01-31 | Obsidian Therapeutics, Inc. | Von pde5 abgeleitete regulatorische konstrukte und verfahren zur verwendung in der immuntherapie |
US20210386788A1 (en) | 2018-10-24 | 2021-12-16 | Obsidian Therapeutics, Inc. | Er tunable protein regulation |
WO2020171889A1 (en) | 2019-02-19 | 2020-08-27 | University Of Rochester | Blocking lipid accumulation or inflammation in thyroid eye disease |
EP3935159A1 (de) | 2019-03-08 | 2022-01-12 | Obsidian Therapeutics, Inc. | Menschliche carboanhydrase-2-zusammensetzungen und verfahren zur abstimmbaren regulierung |
WO2020209934A1 (en) | 2019-04-11 | 2020-10-15 | Massachusetts Institute Of Technology | Novel chimeric antigen receptors and libraries |
CN114126666A (zh) | 2019-04-28 | 2022-03-01 | 西莱克塔生物科技公司 | 用于治疗针对病毒转移载体具有预先存在的免疫力的对象的方法 |
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BR112021023594A2 (pt) | 2019-05-28 | 2022-02-08 | Selecta Biosciences Inc | Métodos e composições para resposta imune de vetor de transferência antiviral atenuada |
US20230092895A1 (en) | 2019-08-30 | 2023-03-23 | Obsidian Therapeutics, Inc. | Tandem cd19 car-based compositions and methods for immunotherapy |
WO2021046451A1 (en) | 2019-09-06 | 2021-03-11 | Obsidian Therapeutics, Inc. | Compositions and methods for dhfr tunable protein regulation |
CN113046392B (zh) * | 2019-12-27 | 2023-04-07 | 深圳市禾沐基因生物技术有限责任公司 | 治疗β-球蛋白功能缺失的重组慢病毒载体及制备方法和应用 |
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CA3227108A1 (en) | 2021-08-11 | 2023-02-16 | Xiaomeng HU | Genetically modified primary cells for allogeneic cell therapy |
WO2023047089A1 (en) | 2021-09-21 | 2023-03-30 | Oslo Universitetssykehus Hf | Binding proteins for terminal deoxynucleotidyl transferase (tdt) |
WO2023064367A1 (en) | 2021-10-12 | 2023-04-20 | Selecta Biosciences, Inc. | Methods and compositions for attenuating anti-viral transfer vector igm responses |
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AU6498294A (en) * | 1993-04-07 | 1994-10-24 | Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The | Dna sequence which acts as a chromatin insulator element to protect expressed genes from (cis)-acting regulatory sequences in mammalian cells |
EP0783575B1 (de) * | 1994-09-19 | 1999-06-16 | Massachusetts Institute Of Technology | Anti-sickling beta-globin,zusammensetzung und verfahren zur behandlung von sichelzellanämie |
WO1998017684A2 (en) * | 1996-10-25 | 1998-04-30 | Advanced Research & Technology Institute | Dna sequences encoding fusions of dna repair proteins and uses thereof |
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- 2003-12-11 AT AT03813391T patent/ATE474460T1/de not_active IP Right Cessation
- 2003-12-11 WO PCT/US2003/039526 patent/WO2004054512A2/en not_active Application Discontinuation
- 2003-12-11 AU AU2003296958A patent/AU2003296958A1/en not_active Abandoned
- 2003-12-11 DE DE60333487T patent/DE60333487D1/de not_active Expired - Lifetime
- 2003-12-11 ES ES03813391T patent/ES2348868T3/es not_active Expired - Lifetime
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US11701435B2 (en) | 2023-07-18 |
US20120009161A1 (en) | 2012-01-12 |
AU2003296958A8 (en) | 2004-07-09 |
HK1082379A1 (en) | 2006-06-09 |
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WO2004054512A3 (en) | 2005-01-06 |
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EP1581056A4 (de) | 2007-01-17 |
US20110274669A1 (en) | 2011-11-10 |
EP1581056B1 (de) | 2010-07-21 |
CY1112327T1 (el) | 2015-12-09 |
AU2003296958A1 (en) | 2004-07-09 |
US9068199B2 (en) | 2015-06-30 |
EP1581056A2 (de) | 2005-10-05 |
US20190038775A1 (en) | 2019-02-07 |
US7901671B2 (en) | 2011-03-08 |
ES2348868T3 (es) | 2010-12-16 |
US20060057725A1 (en) | 2006-03-16 |
US20160022839A1 (en) | 2016-01-28 |
WO2004054512A2 (en) | 2004-07-01 |
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