ES2759049T3 - Vectores retrovirales - Google Patents

Vectores retrovirales Download PDF

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Publication number
ES2759049T3
ES2759049T3 ES14787036T ES14787036T ES2759049T3 ES 2759049 T3 ES2759049 T3 ES 2759049T3 ES 14787036 T ES14787036 T ES 14787036T ES 14787036 T ES14787036 T ES 14787036T ES 2759049 T3 ES2759049 T3 ES 2759049T3
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Spain
Prior art keywords
vector
ltr
rna
pbs
rna packaging
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Active
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ES14787036T
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English (en)
Spanish (es)
Inventor
Conrad Vink
Steven Howe
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UCL Business Ltd
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UCL Business Ltd
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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/8509Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/15011Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/16011Human Immunodeficiency Virus, HIV
    • C12N2740/16022New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/16011Human Immunodeficiency Virus, HIV
    • C12N2740/16041Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/16011Human Immunodeficiency Virus, HIV
    • C12N2740/16051Methods of production or purification of viral material
    • C12N2740/16052Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/48Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
ES14787036T 2013-10-16 2014-10-16 Vectores retrovirales Active ES2759049T3 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GBGB1318347.0A GB201318347D0 (en) 2013-10-16 2013-10-16 Retroviral vectors
PCT/GB2014/053102 WO2015056014A1 (en) 2013-10-16 2014-10-16 Retroviral vectors

Publications (1)

Publication Number Publication Date
ES2759049T3 true ES2759049T3 (es) 2020-05-07

Family

ID=49680159

Family Applications (1)

Application Number Title Priority Date Filing Date
ES14787036T Active ES2759049T3 (es) 2013-10-16 2014-10-16 Vectores retrovirales

Country Status (7)

Country Link
US (1) US10184136B2 (enExample)
EP (1) EP3058076B1 (enExample)
JP (1) JP6525986B2 (enExample)
CN (1) CN105705645B (enExample)
ES (1) ES2759049T3 (enExample)
GB (1) GB201318347D0 (enExample)
WO (1) WO2015056014A1 (enExample)

Families Citing this family (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3443001B1 (en) 2016-04-11 2025-04-30 Obsidian Therapeutics, Inc. REGULATED BIOCIRCUIT SYSTEMS
PT3445406T (pt) 2016-04-20 2021-08-30 Fundacion Instituto De Investig Sanitaria Fundacion Jimenez Diaz Composições e métodos para potenciar a expressão de gene de pklr
SG11201901718VA (en) * 2016-09-08 2019-03-28 Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas Gene therapy for patients with fanconi anemia
US12060566B2 (en) * 2017-08-22 2024-08-13 The Regents Of The University Of California Self-inactivating lentiviral vector comprising a FOXP3 expression cassette
US11642422B2 (en) 2017-10-16 2023-05-09 Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, O.A, M.P. Lentiviral vectors for delivery of PKLR to treat pyruvate kinase deficiency
MX2020011386A (es) 2018-04-27 2021-01-29 Spacecraft Seven Llc Terapia genica para la degeneracion del snc.
CN108676815A (zh) * 2018-05-31 2018-10-19 深圳市免疫基因治疗研究院 一种b型血友病慢病毒载体、慢病毒及其制备方法和应用
US20220403001A1 (en) 2018-06-12 2022-12-22 Obsidian Therapeutics, Inc. Pde5 derived regulatory constructs and methods of use in immunotherapy
EP3870600A1 (en) 2018-10-24 2021-09-01 Obsidian Therapeutics, Inc. Er tunable protein regulation
KR20210149251A (ko) 2019-03-08 2021-12-08 옵시디안 테라퓨틱스, 인크. 조율가능한 조절을 위한 인간 탄산 무수화효소 2 조성물 및 방법
EP3966227A1 (en) 2019-05-07 2022-03-16 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
WO2020252404A1 (en) 2019-06-12 2020-12-17 Obsidian Therapeutics, Inc. Ca2 compositions and methods for tunable regulation
JP2022537670A (ja) 2019-06-12 2022-08-29 オブシディアン セラピューティクス, インコーポレイテッド Ca2の組成物および調整可能な制御方法
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
AU2021320902A1 (en) 2020-08-07 2023-04-06 Spacecraft Seven, Llc Plakophilin-2 (PKP2) gene therapy using AAV vector
GB202019084D0 (en) * 2020-12-03 2021-01-20 Ucl Business Ltd Retroviral vectors
CN117043337A (zh) * 2021-01-25 2023-11-10 普利维尔治疗公司 基于aav的基因表达的调节
AU2022301302A1 (en) 2021-07-01 2024-01-25 Indapta Therapeutics, Inc. Engineered natural killer (nk) cells and related methods
EP4463135A2 (en) 2022-01-10 2024-11-20 Sana Biotechnology, Inc. Methods of ex vivo dosing and administration of lipid particles or viral vectors and related systems and uses
US20250144235A1 (en) 2022-02-02 2025-05-08 Sana Biotechnology, Inc. Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses
CN114931652B (zh) * 2022-06-23 2024-01-26 中国人民解放军陆军军医大学第一附属医院 一种Hmga2基因在修复受损视网膜中的应用
JP2025525439A (ja) 2022-06-30 2025-08-05 インダプタ セラピューティクス インコーポレイテッド 操作されたナチュラルキラー(nk)細胞と抗体療法との組み合わせおよび関連する方法
WO2024026377A1 (en) 2022-07-27 2024-02-01 Sana Biotechnology, Inc. Methods of transduction using a viral vector and inhibitors of antiviral restriction factors
GB202306491D0 (en) 2023-05-03 2023-06-14 Ucl Business Ltd Retroviral vectors

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR2707091B1 (fr) * 1993-06-30 1997-04-04 Cohen Haguenauer Odile Vecteur rétroviral pour le transfert et l'expression de gènes dans des cellules eucaryotes.
US5714353A (en) * 1994-05-24 1998-02-03 Research Corporation Technologies, Inc. Safe vectors for gene therapy
FR2751345B1 (fr) 1996-07-16 1998-09-18 Univ Paris Curie Lignees d'encapsidation hautement productrices
US20050266544A1 (en) 1999-04-30 2005-12-01 Human Gene Therapy Research Institute Methods and compositions for increasing viral vector production in packaging cell lines
US8158413B2 (en) * 2005-10-17 2012-04-17 Institut Pasteur Lentiviral vector-based vaccine
JP2007054069A (ja) * 2005-08-24 2007-03-08 Medizinische Hochschule Hannover 自己不活性化レトロウイルスベクター
CN102443604B (zh) * 2011-11-25 2014-12-31 上海市儿童医院 一种安全性慢病毒载体及其应用

Also Published As

Publication number Publication date
JP6525986B2 (ja) 2019-06-05
CN105705645A (zh) 2016-06-22
CN105705645B (zh) 2019-11-15
GB201318347D0 (en) 2013-11-27
EP3058076A1 (en) 2016-08-24
JP2016539629A (ja) 2016-12-22
EP3058076B1 (en) 2019-09-18
US20160230191A1 (en) 2016-08-11
US10184136B2 (en) 2019-01-22
WO2015056014A1 (en) 2015-04-23

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