ATE474460T1 - Therapeutische retrovirus-vektoren für gentherapie - Google Patents
Therapeutische retrovirus-vektoren für gentherapieInfo
- Publication number
- ATE474460T1 ATE474460T1 AT03813391T AT03813391T ATE474460T1 AT E474460 T1 ATE474460 T1 AT E474460T1 AT 03813391 T AT03813391 T AT 03813391T AT 03813391 T AT03813391 T AT 03813391T AT E474460 T1 ATE474460 T1 AT E474460T1
- Authority
- AT
- Austria
- Prior art keywords
- gene therapy
- hematopoietic
- retrovirus vectors
- cells
- methods
- Prior art date
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
-
- 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
-
- 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; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
- A01K67/027—New or modified breeds of vertebrates
- A01K67/0275—Genetically modified vertebrates, e.g. transgenic
- A01K67/0278—Knock-in vertebrates, e.g. humanised vertebrates
-
- 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
- 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; AVICULTURE; APICULTURE; PISCICULTURE; 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; AVICULTURE; APICULTURE; PISCICULTURE; 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; AVICULTURE; APICULTURE; PISCICULTURE; 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; AVICULTURE; APICULTURE; PISCICULTURE; 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Biophysics (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Environmental Sciences (AREA)
- Virology (AREA)
- Immunology (AREA)
- Diabetes (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Hematology (AREA)
- General Chemical & Material Sciences (AREA)
- Animal Husbandry (AREA)
- Biodiversity & Conservation Biology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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 |
---|---|
ATE474460T1 true ATE474460T1 (de) | 2010-08-15 |
Family
ID=32600914
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
AT03813391T ATE474460T1 (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 (70)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
DE60333487D1 (de) * | 2002-12-13 | 2010-09-02 | Genetix Pharmaceuticals Inc | Therapeutische retrovirus-vektoren für gentherapie |
US8921050B2 (en) | 2006-03-17 | 2014-12-30 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Methods of diagnosing renal cell carcinoma |
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 |
EP3214174B1 (de) | 2010-03-04 | 2019-10-16 | InteRNA Technologies B.V. | Mirna-molekül, das über seine quelle definiert ist, und seine diagnostischen und therapeutischen verwendungen bei mit emt assoziierten krankheiten |
AU2011274619B2 (en) | 2010-07-06 | 2016-11-10 | 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 |
KR20150029756A (ko) | 2011-06-10 | 2015-03-18 | 블루버드 바이오, 인코포레이티드. | 부신백질이영양증 및 부신척수신경병증을 위한 유전자 요법 벡터 |
BR112014007029A2 (pt) | 2011-09-23 | 2017-04-11 | Bluebird Bio Inc | métodos aperfeiçoados de terapia gênica |
SG10201602423TA (en) | 2011-09-30 | 2016-05-30 | Bluebird Bio Inc | Compounds For Improved Viral Transduction |
ES2688619T3 (es) | 2011-12-22 | 2018-11-05 | Interna Technologies B.V. | MiARN para tratar el cáncer de cabeza y de cuello |
EP4052717A1 (de) * | 2012-03-13 | 2022-09-07 | Celularity Inc. | Modifizierte erythrozytenvorläuferzellen und verwendungen davon |
JP2015529466A (ja) * | 2012-09-14 | 2015-10-08 | 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 | 鎌状赤血球症の幹細胞遺伝子治療のためのレンチウイルスベクター |
WO2014072357A1 (en) | 2012-11-06 | 2014-05-15 | Interna Technologies B.V. | Combination for use in treating diseases or conditions associated with melanoma, or treating diseases or conditions associated with activated b-raf pathway |
SG10201704008RA (en) | 2012-11-27 | 2017-06-29 | The Children's Medical Center Corp | Targeting Bcl11a Distal Regulatory Elements for Fetal Hemoglobin Reinduction |
EP3071515A2 (de) | 2013-11-18 | 2016-09-28 | Rubius Therapeutics, Inc. | Synthetische membran-empfänger-komplexe |
HUE054471T2 (hu) | 2014-04-01 | 2021-09-28 | Rubius Therapeutics Inc | Immunmodulációs módszerek és készítmények |
CA2946309C (en) | 2014-04-25 | 2021-11-09 | Michael MILSOM | Synthetic bcl11a micrornas for treating hemoglobinopathies |
WO2016033201A1 (en) * | 2014-08-26 | 2016-03-03 | The Board Of Trustees Of The Leland Stanford Junior University | Engraftment of stem cells with a combination of an agent that targets stem cells and modulation of immunoregulatory signaling |
CN107073091A (zh) | 2014-09-07 | 2017-08-18 | 西莱克塔生物科技公司 | 用于减弱外显子跳读抗病毒转移载体免疫应答的方法和组合物 |
AU2015315726B2 (en) * | 2014-09-11 | 2021-08-19 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Lentiviral vector for treating hemoglobin disorders |
EP3247409B1 (de) * | 2015-01-21 | 2020-11-04 | Cornell University | Virale vektoren zur prophylaxe und therapie von hämoglobinopathien |
WO2016182917A1 (en) | 2015-05-08 | 2016-11-17 | Children's Medical Center Corporation | Targeting bcl11a enhancer functional regions for fetal hemoglobin reinduction |
GB201600328D0 (en) | 2016-01-08 | 2016-02-24 | Univ Oslo Hf | Anti-CD37 chimeric antigen receptors and immune cells expressing them |
WO2017139561A1 (en) | 2016-02-12 | 2017-08-17 | Bluebird Bio, Inc. | Vcn enhancer compositions and methods of using the same |
CA3014078A1 (en) | 2016-02-12 | 2017-08-17 | Bluebird Bio, Inc. | Vcn enhancer compositions and methods of using the same |
US11446398B2 (en) | 2016-04-11 | 2022-09-20 | Obsidian Therapeutics, Inc. | Regulated biocircuit systems |
JP2019535839A (ja) | 2016-11-29 | 2019-12-12 | ピュアテック ヘルス エルエルシー | 治療剤の送達のためのエクソソーム |
EP3548617A4 (de) | 2016-12-05 | 2020-06-17 | The Regents of The University of California | Optimierter lentiviraler vektor zur stammzellengentherapie von hämoglobinopathien |
AU2018205496A1 (en) | 2017-01-07 | 2019-07-25 | Selecta Biosciences, Inc. | Patterned dosing of immunosuppressants coupled to synthetic nanocarriers |
US20190367944A1 (en) | 2017-01-30 | 2019-12-05 | The U.S.A., As Represented By The Secretary, Department Of Health And Human Services | Recombinant virus vectors for the treatment of glycogen storage disease |
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 |
EP3684421A4 (de) | 2017-09-18 | 2021-08-04 | Children's Hospital Medical Center | Starker isolator und seine verwendung im gentransfer |
JP7427584B2 (ja) | 2017-10-13 | 2024-02-05 | セレクタ バイオサイエンシーズ インコーポレーテッド | 抗ウイルス導入ベクターigm応答を減弱化するための方法および組成物 |
CN111566212A (zh) | 2017-11-03 | 2020-08-21 | 因特尔纳技术有限公司 | miRNA分子,等同物,安塔够妙或其来源用于治疗和/或诊断与神经元缺陷相关的病症和/或疾病或用于神经元生成和/或再生 |
JP2021515037A (ja) | 2018-02-26 | 2021-06-17 | アントルクス,インコーポレーテッド | 寛容原性リポソーム及びその使用方法 |
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 |
US20220403001A1 (en) | 2018-06-12 | 2022-12-22 | Obsidian Therapeutics, Inc. | Pde5 derived regulatory constructs and methods of use in immunotherapy |
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 |
CA3132840A1 (en) | 2019-03-08 | 2020-09-17 | Obsidian Therapeutics, Inc. | Human carbonic anhydrase 2 compositions and methods for tunable regulation |
WO2020209934A1 (en) | 2019-04-11 | 2020-10-15 | Massachusetts Institute Of Technology | Novel chimeric antigen receptors and libraries |
JP2022531201A (ja) | 2019-04-28 | 2022-07-06 | セレクタ バイオサイエンシーズ インコーポレーテッド | ウイルス導入ベクターに対する既存の免疫を伴う対象の処置のための方法 |
US20240124889A1 (en) | 2019-05-07 | 2024-04-18 | Voyager Therapeutics, Inc. | Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation |
WO2020236263A1 (en) | 2019-05-23 | 2020-11-26 | Massachusetts Institute Of Technology | Ligand discovery and gene delivery via retroviral surface display |
BR112021023594A2 (pt) | 2019-05-28 | 2022-02-08 | Selecta Biosciences Inc | Métodos e composições para resposta imune de vetor de transferência antiviral atenuada |
WO2021040736A1 (en) | 2019-08-30 | 2021-03-04 | 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 |
WO2022235929A1 (en) | 2021-05-05 | 2022-11-10 | Radius Pharmaceuticals, Inc. | Animal model having homologous recombination of mouse pth1 receptor |
KR20240051112A (ko) | 2021-07-01 | 2024-04-19 | 인답타 세라뷰틱스 인코포레이티드 | 조작된 자연 살해(nk) 세포 및 관련 방법 |
KR20240035852A (ko) | 2021-07-19 | 2024-03-18 | 2세븐티 바이오, 인코포레이티드 | 벡터 제조 프로세스 |
AU2022325955A1 (en) | 2021-08-11 | 2024-02-08 | Sana Biotechnology, Inc. | Genetically modified cells for allogeneic cell therapy to reduce instant blood mediated inflammatory reactions |
CA3227108A1 (en) | 2021-08-11 | 2023-02-16 | Xiaomeng HU | Genetically modified primary cells for allogeneic cell therapy |
AU2022325231A1 (en) | 2021-08-11 | 2024-02-08 | Sana Biotechnology, Inc. | Genetically modified cells for allogeneic cell therapy to reduce complement-mediated inflammatory reactions |
AU2022326565A1 (en) | 2021-08-11 | 2024-02-08 | Sana Biotechnology, Inc. | Genetically modified cells for allogeneic cell therapy |
WO2023047089A1 (en) | 2021-09-21 | 2023-03-30 | Oslo Universitetssykehus Hf | Binding proteins for terminal deoxynucleotidyl transferase (tdt) |
US20230141563A1 (en) | 2021-10-12 | 2023-05-11 | Selecta Biosciences, Inc. | Methods and compositions for attenuating anti-viral transfer vector igm responses |
WO2023114918A1 (en) | 2021-12-16 | 2023-06-22 | Ludwig Institute For Cancer Research Ltd | Antisense transfer vectors and methods of use thereof |
WO2023133595A2 (en) | 2022-01-10 | 2023-07-13 | Sana Biotechnology, Inc. | Methods of ex vivo dosing and administration of lipid particles or viral vectors and related systems and uses |
WO2023150647A1 (en) | 2022-02-02 | 2023-08-10 | Sana Biotechnology, Inc. | Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses |
WO2023158836A1 (en) | 2022-02-17 | 2023-08-24 | Sana Biotechnology, Inc. | Engineered cd47 proteins and uses thereof |
US20230357437A1 (en) | 2022-03-09 | 2023-11-09 | Selecta Biosciences, Inc. | Immunosuppressants in combination with anti-igm agents and related dosing |
WO2023173123A1 (en) | 2022-03-11 | 2023-09-14 | Sana Biotechnology, Inc. | Genetically modified cells and compositions and uses thereof |
CN114457119B (zh) * | 2022-04-11 | 2022-08-12 | 中吉智药(南京)生物技术有限公司 | 慢病毒载体在制备治疗β-地中海贫血药物中的应用 |
WO2024007020A1 (en) | 2022-06-30 | 2024-01-04 | Indapta Therapeutics, Inc. | Combination of engineered natural killer (nk) cells and antibody therapy and related methods |
WO2024026377A1 (en) | 2022-07-27 | 2024-02-01 | Sana Biotechnology, Inc. | Methods of transduction using a viral vector and inhibitors of antiviral restriction factors |
WO2024050450A1 (en) | 2022-08-31 | 2024-03-07 | Gigamune, Inc. | Engineered enveloped vectors and methods of use thereof |
Family Cites Families (11)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO1994023046A1 (en) * | 1993-04-07 | 1994-10-13 | The Government Of The United States Of America As Represented By The Secretary, Department Of Health And Human Services | Dna sequence which acts as a chromatin insulator element to protect expressed genes from cis-acting regulatory sequences in mammalian cells |
CA2199832C (en) * | 1994-09-19 | 2000-08-01 | Philippe Leboulch | Anti-sickling beta-globin protein, compositions and methods for treating sickle cell disease |
US6046036A (en) * | 1996-10-25 | 2000-04-04 | Advanced Research And Technology Institute | DNA sequences encoding fusions of DNA repair proteins and uses thereof |
US6531123B1 (en) * | 1997-05-01 | 2003-03-11 | Lung-Ji Chang | Lentiviral vectors |
US6136597A (en) * | 1997-09-18 | 2000-10-24 | The Salk Institute For Biological Studies | RNA export element |
US6395549B1 (en) * | 1998-10-22 | 2002-05-28 | Medical College Of Georgia Research Institute, Inc. | Long terminal repeat, enhancer, and insulator sequences for use in recombinant vectors |
WO2002083080A2 (en) | 2001-04-17 | 2002-10-24 | Genetix Pharmaceuticals, Inc. | Method of treating arthritis using lentiviral vectors in gene therapy |
US20040033604A1 (en) | 2001-04-20 | 2004-02-19 | Gary Kobinger | Recombinant lentiviral vectors pseudotyped in envelopes containing filovirus binding domains |
WO2002087341A1 (en) * | 2001-05-01 | 2002-11-07 | Genetix Pharmaceuticals, Inc. | Novel self-inactivating (sin) lentiviral vectors |
NZ532060A (en) * | 2001-10-02 | 2005-11-25 | Inst Clayton De La Rech | Methods and compositions relating to restricted expression lentiviral vectors and their applications |
DE60333487D1 (de) | 2002-12-13 | 2010-09-02 | Genetix Pharmaceuticals Inc | Therapeutische retrovirus-vektoren für gentherapie |
-
2003
- 2003-12-11 DE DE60333487T patent/DE60333487D1/de not_active Expired - Lifetime
- 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 EP EP03813391A patent/EP1581056B1/de not_active Expired - Lifetime
- 2003-12-11 AU AU2003296958A patent/AU2003296958A1/en not_active Abandoned
- 2003-12-11 ES ES03813391T patent/ES2348868T3/es not_active Expired - Lifetime
-
2005
- 2005-06-09 US US11/150,785 patent/US7901671B2/en not_active Expired - Lifetime
-
2006
- 2006-04-04 HK HK06104166.4A patent/HK1082379A1/xx not_active IP Right Cessation
-
2010
- 2010-10-11 CY CY20101100906T patent/CY1112327T1/el unknown
- 2010-12-22 US US12/976,078 patent/US20110274669A1/en not_active Abandoned
-
2011
- 2011-09-23 US US13/242,815 patent/US9068199B2/en not_active Expired - Lifetime
-
2015
- 2015-04-29 US US14/700,021 patent/US10105451B2/en not_active Expired - Lifetime
-
2018
- 2018-09-13 US US16/130,845 patent/US10632212B2/en not_active Expired - Lifetime
-
2020
- 2020-03-16 US US16/820,066 patent/US11701435B2/en active Active
Also Published As
Publication number | Publication date |
---|---|
AU2003296958A1 (en) | 2004-07-09 |
HK1082379A1 (en) | 2006-06-09 |
WO2004054512A3 (en) | 2005-01-06 |
US9068199B2 (en) | 2015-06-30 |
WO2004054512A2 (en) | 2004-07-01 |
US10105451B2 (en) | 2018-10-23 |
US20190038775A1 (en) | 2019-02-07 |
US20160022839A1 (en) | 2016-01-28 |
US20200368370A1 (en) | 2020-11-26 |
EP1581056A4 (de) | 2007-01-17 |
US20060057725A1 (en) | 2006-03-16 |
US7901671B2 (en) | 2011-03-08 |
EP1581056B1 (de) | 2010-07-21 |
US20120009161A1 (en) | 2012-01-12 |
AU2003296958A8 (en) | 2004-07-09 |
US20110274669A1 (en) | 2011-11-10 |
US10632212B2 (en) | 2020-04-28 |
EP1581056A2 (de) | 2005-10-05 |
US11701435B2 (en) | 2023-07-18 |
DE60333487D1 (de) | 2010-09-02 |
ES2348868T3 (es) | 2010-12-16 |
CY1112327T1 (el) | 2015-12-09 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
ATE474460T1 (de) | Therapeutische retrovirus-vektoren für gentherapie | |
MX344587B (es) | Metodo para incrementar la estabilidad in vitro e in vivo de enzimas asociadas al transtorno lisosomal. | |
WO2002064748A8 (en) | Multipotent adult stem cells, sources thereof, methods of obtaining and maintaining same, methods of differentiation thereof, methods of use thereof and cells derived thereof | |
AU7736300A (en) | Therapeutic treatment of androgen receptor driven conditions | |
AU2003303384A8 (en) | Uses of mammalian cytokine; related reagents | |
ATE524554T1 (de) | Nicht integrativer und nicht replikativer lentivirus, herstellung und verwendungen | |
DE60231439D1 (de) | Mitotische kinesinhemmer | |
MX2007004551A (es) | Compuestos tiadiazol y metodos de uso. | |
WO2006092668A3 (en) | Fetal skin cell protein compositions for the treatment of skin conditions, disorders or diseases and methods of making and using the same | |
DE60222302D1 (de) | Inhibitoren von mitotischem kinesin | |
DE60324449D1 (de) | Inhibitoren der phosphodiesterase der zyklischen n, und deren verwendung in therapie | |
ATE211392T1 (de) | Verwendung des glialen, neurotrophen faktors (gdnf) zur behandlung von gehörstörungen | |
SG166672A1 (en) | Methods of down regulating target gene expression in vivo by introduction of interfering rna | |
WO2005049000A3 (en) | Treatment of phenylketonurias with bh4 | |
PL356846A1 (en) | Use of interleukin-6 antagonists for the treatment of diseases characterised by high levels of aromatase | |
TW200640944A (en) | VGF polypeptides and methods of treating VGF-related disorders | |
DK1274464T3 (da) | Sammensætninger og fremgangsmåder til regulering af proteinekspression i tarm | |
TR200102985T2 (tr) | Gen tedavisi teknikleri. | |
ATE321566T1 (de) | Verwendung von long pentraxin ptx3 für die behandlung von infertilität bei frauen | |
DK1744736T3 (da) | Fremgangsmåde til behandling af problemer med tørre øjne og uveitis | |
ATE287722T1 (de) | Mittel zur behandlung maligner erkrankungen unter verwendung des proteins yb-1 | |
MXPA02000680A (es) | Polipeptidos vgf y metodos para tratar desordenes relacionados con vgf. | |
HK1074840A1 (en) | Novel dithiolopyrrolones with therapeutic activity | |
TW200503693A (en) | 4-, 6- or 7-hydroxyindoles with n-oxide groups and the use thereof as therapeutic agents | |
MX2021014028A (es) | Métodos y composiciones para tratar obesidad y/o trastornos de la piel. |
Legal Events
Date | Code | Title | Description |
---|---|---|---|
RER | Ceased as to paragraph 5 lit. 3 law introducing patent treaties |