IN2014CN02518A - - Google Patents
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- Publication number
- IN2014CN02518A IN2014CN02518A IN2518CHN2014A IN2014CN02518A IN 2014CN02518 A IN2014CN02518 A IN 2014CN02518A IN 2518CHN2014 A IN2518CHN2014 A IN 2518CHN2014A IN 2014CN02518 A IN2014CN02518 A IN 2014CN02518A
- Authority
- IN
- India
- Prior art keywords
- methods
- cells
- compositions
- hematopoietic stem
- present
- Prior art date
Links
- 238000000034 method Methods 0.000 abstract 4
- 210000003958 hematopoietic stem cell Anatomy 0.000 abstract 3
- 210000004027 cell Anatomy 0.000 abstract 2
- 239000000203 mixture Substances 0.000 abstract 2
- 241000700605 Viruses Species 0.000 abstract 1
- 238000001415 gene therapy Methods 0.000 abstract 1
- 239000000463 material Substances 0.000 abstract 1
- 230000001225 therapeutic effect Effects 0.000 abstract 1
- 230000002463 transducing effect Effects 0.000 abstract 1
- 230000026683 transduction Effects 0.000 abstract 1
- 238000010361 transduction Methods 0.000 abstract 1
- 239000013603 viral vector Substances 0.000 abstract 1
- 230000003612 virological effect Effects 0.000 abstract 1
Classifications
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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
-
- 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)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P5/00—Drugs for disorders of the endocrine system
- A61P5/38—Drugs for disorders of the endocrine system of the suprarenal hormones
-
- 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
- A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
-
- 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
- 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
- C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K2035/124—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells the cells being hematopoietic, bone marrow derived or blood 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
- 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
- C12N2501/00—Active agents used in cell culture processes, e.g. differentation
- C12N2501/02—Compounds of the arachidonic acid pathway, e.g. prostaglandins, leukotrienes
-
- 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
- C12N2740/16011—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
- C12N2810/00—Vectors comprising a targeting moiety
- C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
- C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
- C12N2810/6072—Vectors comprising as targeting moiety peptide derived from defined protein from viruses negative strand RNA viruses
- C12N2810/6081—Vectors comprising as targeting moiety peptide derived from defined protein from viruses negative strand RNA viruses rhabdoviridae, e.g. VSV
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Hematology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Immunology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Diabetes (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Biophysics (AREA)
- Molecular Biology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Epidemiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Cell Biology (AREA)
- Developmental Biology & Embryology (AREA)
- Endocrinology (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 (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201161541736P | 2011-09-30 | 2011-09-30 | |
| PCT/US2012/057987 WO2013049615A1 (en) | 2011-09-30 | 2012-09-28 | Compounds for improved viral transduction |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IN2014CN02518A true IN2014CN02518A (cg-RX-API-DMAC7.html) | 2015-07-31 |
Family
ID=47996444
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IN2518CHN2014 IN2014CN02518A (cg-RX-API-DMAC7.html) | 2011-09-30 | 2012-09-28 |
Country Status (19)
Families Citing this family (50)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2845641T3 (es) | 2008-11-06 | 2021-07-27 | Univ Indiana Res & Tech Corp | Materiales y métodos para mejorar procedimientos de injerto funcionante de células madre hematopoyéticas |
| DK3269802T3 (da) * | 2011-09-30 | 2020-02-03 | Bluebird Bio Inc | Forbindelser til forbedret virustransduktion |
| ES2713988T3 (es) | 2012-08-01 | 2019-05-24 | United Therapeutics Corp | Tratamiento de hipertensión arterial pulmonar con células progenitoras endoteliales tratadas con prostaciclina |
| CA2880808C (en) | 2012-08-01 | 2023-01-24 | United Therapeutics Corporation | Treatment of pulmonary arterial hypertension with mesenchymal stem cells |
| WO2014026110A2 (en) * | 2012-08-10 | 2014-02-13 | Bluebird Bio, Inc. | Compounds for improved viral transduction |
| MX361412B (es) | 2012-11-27 | 2018-12-05 | Childrens Medical Center | Direccion de elementos reguladores distales de bcl11a para reinduccion de hemoglobina fetal. |
| ES2963968T3 (es) | 2013-01-09 | 2024-04-03 | United Therapeutics Corp | Tratamiento de la vasculopatía con prostaciclina y células madre mesenquimatosas |
| SG11201603166UA (en) * | 2013-10-24 | 2016-05-30 | Ospedale San Raffaele Srl | Method |
| EP3122181A4 (en) * | 2014-03-26 | 2018-04-25 | The Brigham and Women's Hospital, Inc. | Compositions and methods for ex vivo expansion of human hematopoietic stem/progenitor cells |
| BR112016024565A2 (pt) | 2014-04-25 | 2018-01-23 | Children's Medical Center Corporation | composições e métodos de tratar hemoglobinopatias |
| EP3763814A1 (en) | 2015-05-08 | 2021-01-13 | The Children's Medical Center Corporation | Targeting bcl11a enhancer functional regions for fetal hemoglobin reinduction |
| RU2630654C2 (ru) * | 2015-07-29 | 2017-09-11 | Федеральное государственное автономное образовательное учреждение высшего профессионального образования "Балтийский Федеральный Университет имени Иммануила Канта" (БФУ им. И. Канта) | Способ хемогенетической регистрации и коррекции нейрогенеза на основе генетических конструкций для трансфекции астроцитов и нейронов |
| CN105062979A (zh) * | 2015-08-21 | 2015-11-18 | 昆明理工大学 | 一种培养戊型肝炎病毒的方法 |
| CN108883136A (zh) * | 2016-02-12 | 2018-11-23 | 蓝鸟生物公司 | Vcn增强子组合物及其使用方法 |
| EP4151720B1 (en) * | 2016-02-12 | 2024-08-07 | bluebird bio, Inc. | Vcn enhancer compositions and methods of using the same |
| AU2017248259A1 (en) * | 2016-04-07 | 2018-10-25 | Bluebird Bio, Inc. | Chimeric antigen receptor T cell compositions |
| JP2019519489A (ja) | 2016-05-05 | 2019-07-11 | リクイディア・テクノロジーズ・インコーポレーテッド | 肺高血圧症を治療するための乾燥粉末トレプロスチニル |
| CA3041514A1 (en) | 2016-10-24 | 2018-05-03 | United Therapeutics Corporation | Enhancement of msc immunomodulatory properties by treprostinil |
| EP3548005A4 (en) | 2016-11-29 | 2020-06-17 | Puretech Health LLC | Exosomes for delivery of therapeutic agents |
| US11261441B2 (en) | 2017-03-29 | 2022-03-01 | Bluebird Bio, Inc. | Vectors and compositions for treating hemoglobinopathies |
| CA3057862A1 (en) * | 2017-03-29 | 2018-10-04 | Bluebird Bio, Inc. | Vectors and compositions for treating hemoglobinopathies |
| US11788087B2 (en) | 2017-05-25 | 2023-10-17 | The Children's Medical Center Corporation | BCL11A guide delivery |
| WO2019055814A2 (en) * | 2017-09-15 | 2019-03-21 | The Medical College Of Wisconsin, Inc. | EPOXYEICOSATRIENOIC ACID (EET) ANALOGUES TARGETING KIDNEYS |
| FR3074189A1 (fr) * | 2017-11-30 | 2019-05-31 | Assistance Publique - Hopitaux De Paris | Methode de generation de greffons de cellules souches hematopoietiques |
| CN110799641B (zh) * | 2018-04-13 | 2021-02-19 | 诺未科技（北京）有限公司 | 丁酸钠的用途及含有丁酸钠的培养体系 |
| CN109207427B (zh) * | 2018-09-17 | 2020-12-08 | 诺未科技（银川）有限公司 | 一种将人造血祖细胞转变为造血干细胞的方法 |
| US12239693B2 (en) | 2018-04-27 | 2025-03-04 | The Medical College Of Wisconsin, Inc. | Use of lentivector-transduced T-Rapa cells for amelioration of lysosomal storage disorders |
| US12350284B2 (en) | 2018-05-02 | 2025-07-08 | The Children's Medical Center Corporation | BCL11A microRNAs for treating hemoglobinopathies |
| US12241079B2 (en) | 2018-09-14 | 2025-03-04 | The Children's Hospital Of Philadelphia | Compositions and methods for hemoglobin production |
| JP2022504722A (ja) * | 2018-10-12 | 2022-01-13 | ライフ テクノロジーズ コーポレーション | 造血幹細胞および造血前駆細胞の増殖システム |
| MA54315A (fr) * | 2018-11-30 | 2021-10-06 | Univ Kyoto | Système d'administration de médicament utilisant une solution |
| WO2020264488A1 (en) * | 2019-06-28 | 2020-12-30 | The Children's Hospital Of Philadelphia | Compositions and methods for treating anemia |
| EP4045666A4 (en) * | 2019-10-16 | 2023-11-15 | Orchard Therapeutics (Europe) Limited | COMPOSITIONS AND METHODS FOR MODIFYING EUKARYOTIC CELLS |
| CN116134136A (zh) * | 2020-06-02 | 2023-05-16 | 康泰伦特药物解决方案有限责任公司 | 具有多个用于基因插入的Dock的细胞系 |
| CN115125270A (zh) * | 2021-03-22 | 2022-09-30 | 华东师范大学 | 一种α-珠蛋白过表达载体及其应用 |
| CN113106098B (zh) * | 2021-04-21 | 2022-04-01 | 贵州医科大学 | 一种在红系细胞中特异表达人β珠蛋白的重组序列及其应用 |
| AU2021202658A1 (en) | 2021-04-28 | 2022-11-17 | Fondazione Telethon | Gene therapy |
| CN113336833A (zh) * | 2021-06-04 | 2021-09-03 | 广州伯尼兹生物科技有限公司 | 一种高效分泌表达猪流行性腹泻病毒s1蛋白的生产方法 |
| US20250082777A1 (en) | 2022-01-10 | 2025-03-13 | Sana Biotechnology, Inc. | Methods of ex vivo dosing and administration of lipid particles or viral vectors and related systems and uses |
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| EP4569092A2 (en) | 2022-08-11 | 2025-06-18 | Fondazione Telethon ETS | Gene therapy of lysosomal storage disorders |
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| WO2024081820A1 (en) | 2022-10-13 | 2024-04-18 | Sana Biotechnology, Inc. | Viral particles targeting hematopoietic stem cells |
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| IT202300011790A1 (it) | 2023-06-08 | 2024-12-08 | Fond Telethon Ets | Protocolli di manipolazione genica in cellule immunitarie |
| WO2025056970A1 (en) * | 2023-09-12 | 2025-03-20 | Virocell Biologics Ltd | Mammalian cell culture additives |
| IT202300022191A1 (it) | 2023-10-24 | 2025-04-24 | Fond Telethon Ets | Mezzi e metodi per la modifica genetica di uba1 |
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| EP4640830A1 (en) | 2024-04-23 | 2025-10-29 | Fondazione Telethon ETS | Means and methods for safe and efficient gene editing in cells |
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