DK2839013T3 - Ikke-disruptiv-gen-targetering - Google Patents
Ikke-disruptiv-gen-targetering Download PDFInfo
- Publication number
- DK2839013T3 DK2839013T3 DK13778431.0T DK13778431T DK2839013T3 DK 2839013 T3 DK2839013 T3 DK 2839013T3 DK 13778431 T DK13778431 T DK 13778431T DK 2839013 T3 DK2839013 T3 DK 2839013T3
- Authority
- DK
- Denmark
- Prior art keywords
- disruptive
- gen
- targeting
- Prior art date
Links
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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian 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
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
-
- 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
- A01K2207/12—Animals modified by administration of exogenous cells
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
- C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
-
- 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
- C12N2840/00—Vectors comprising a special translation-regulating system
- C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Microbiology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Cell Biology (AREA)
- Mycology (AREA)
- Medicinal Chemistry (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
- Pyridine Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261635203P | 2012-04-18 | 2012-04-18 | |
| US201261654645P | 2012-06-01 | 2012-06-01 | |
| PCT/US2013/032443 WO2013158309A2 (en) | 2012-04-18 | 2013-03-15 | Non-disruptive gene targeting |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK2839013T3 true DK2839013T3 (da) | 2020-09-14 |
Family
ID=49380320
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK13778431.0T DK2839013T3 (da) | 2012-04-18 | 2013-03-15 | Ikke-disruptiv-gen-targetering |
Country Status (5)
| Country | Link |
|---|---|
| US (4) | US20130280222A1 (da) |
| EP (2) | EP2839013B1 (da) |
| DK (1) | DK2839013T3 (da) |
| ES (1) | ES2828663T3 (da) |
| WO (1) | WO2013158309A2 (da) |
Families Citing this family (41)
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| EP2854866A4 (en) * | 2012-05-30 | 2015-12-23 | Baylor College Medicine | SUPERHELIC MINI-SECTORS AS A TOOL FOR DNS REPAIR, CHANGE AND REPLACEMENT |
| US10648001B2 (en) | 2012-07-11 | 2020-05-12 | Sangamo Therapeutics, Inc. | Method of treating mucopolysaccharidosis type I or II |
| AU2013289206B2 (en) | 2012-07-11 | 2018-08-09 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of lysosomal storage diseases |
| WO2015068785A1 (ja) * | 2013-11-06 | 2015-05-14 | 国立大学法人広島大学 | 核酸挿入用ベクター |
| EP3119895B1 (en) | 2014-03-21 | 2023-12-20 | The Board of Trustees of the Leland Stanford Junior University | Genome editing without nucleases |
| US20170051354A1 (en) * | 2014-04-28 | 2017-02-23 | Sigma-Aldrich Co. Llc | Epigenetic modification of mammalian genomes using targeted endonucleases |
| US10351825B2 (en) * | 2014-07-11 | 2019-07-16 | The Texas A&M University System | Mesenchymal stem cells derived from induced pluripotent stem cells |
| JP2017534301A (ja) * | 2014-11-10 | 2017-11-24 | マードック チルドレンズ リサーチ インスティチュート | 構成的に発現された遺伝子を含む遺伝子座における標的を狙った組み込みのためのベクター及び方法 |
| US20170369848A1 (en) * | 2014-11-11 | 2017-12-28 | Q Therapeutics, Inc. | Engineering mesenchymal stem cells using homologous recombination |
| US20170362580A1 (en) * | 2014-12-12 | 2017-12-21 | James Zhu | Methods and compositions for selectively eliminating cells of interest |
| EP3250680A4 (en) | 2015-01-26 | 2018-12-05 | Fate Therapeutics, Inc. | Methods and compositions for inducing hematopoietic cell differentiation |
| MY187898A (en) | 2015-03-02 | 2021-10-27 | Adverum Biotechnologies Inc | Compositions and methods for intravitreal delivery of polynucleotides to retinal cones |
| EP3294866A4 (en) * | 2015-05-12 | 2018-12-05 | Sangamo Therapeutics, Inc. | Nuclease-mediated regulation of gene expression |
| WO2017053729A1 (en) * | 2015-09-25 | 2017-03-30 | The Board Of Trustees Of The Leland Stanford Junior University | Nuclease-mediated genome editing of primary cells and enrichment thereof |
| EP3371314B1 (en) | 2015-11-04 | 2023-07-05 | Fate Therapeutics, Inc. | Genomic engineering of pluripotent cells |
| AU2016348342B2 (en) | 2015-11-04 | 2023-07-06 | Fate Therapeutics, Inc. | Methods and compositions for inducing hematopoietic cell differentiation |
| WO2017091512A1 (en) * | 2015-11-23 | 2017-06-01 | Sangamo Biosciences, Inc. | Methods and compositions for engineering immunity |
| EP3443085B1 (en) * | 2016-04-14 | 2022-09-14 | BOCO Silicon Valley, Inc. | Genome editing of human neural stem cells using nucleases |
| PT4180519T (pt) | 2016-04-15 | 2025-09-04 | Memorial Sloan Kettering Cancer Center | Células t transgénicas e composições de células t que expressam um recetor antigénico quimérico e métodos relacionados |
| 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 |
| EP3682004A4 (en) * | 2017-09-15 | 2021-05-26 | The Board of Trustees of the Leland Stanford Junior University | MULTIPLEX PRODUCTION AND BARCODING OF GENETICALLY MODIFIED CELLS |
| BR112020015798A2 (pt) | 2018-02-01 | 2021-03-09 | Homology Medicines, Inc. | Composições de vírus adeno-associado para restaurar função de gene da pah e métodos de uso das mesmas |
| CA3092832A1 (en) * | 2018-03-02 | 2019-09-06 | Generation Bio Co. | Identifying and characterizing genomic safe harbors (gsh) in humans and murine genomes, and viral and non-viral vector compositions for targeted integration at an identified gsh loci |
| AU2018436152A1 (en) | 2018-08-10 | 2021-02-04 | Logicbio Therapeutics, Inc. | Non-disruptive gene therapy for the treatment of MMA |
| CN113166754A (zh) | 2018-10-16 | 2021-07-23 | 蓝色等位基因有限责任公司 | 用于将dna靶向插入基因中的方法 |
| JP7578590B2 (ja) | 2018-10-18 | 2024-11-06 | インテリア セラピューティクス,インコーポレーテッド | 第ix因子を発現するための組成物及び方法 |
| CN113166769A (zh) * | 2018-11-01 | 2021-07-23 | 蓝色等位基因有限责任公司 | 用于改变遗传病症的基因表达的方法 |
| LU101118B1 (en) | 2019-02-06 | 2020-08-06 | Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) | Method for detecting a specific splice event of a gene of interest |
| AU2020279370A1 (en) * | 2019-05-21 | 2021-12-16 | Sangamo Therapeutics, Inc. | Controlled transgene expression in regulatory T cells |
| US11622547B2 (en) | 2019-06-07 | 2023-04-11 | Regeneran Pharmaceuticals, Inc. | Genetically modified mouse that expresses human albumin |
| CA3154998A1 (en) * | 2019-09-17 | 2021-03-25 | Memorial Sloan-Kettering Cancer Center | Methods for identifying genomic safe harbors |
| MX2022005572A (es) | 2019-11-08 | 2022-06-09 | Regeneron Pharma | Estrategias crispr y aav para la terapia de retinosquisis juvenil ligada al cromosoma x. |
| IL297881A (en) * | 2020-05-04 | 2023-01-01 | Editas Medicine Inc | Selection by essential-gene knock-in |
| TW202208632A (zh) | 2020-05-27 | 2022-03-01 | 美商同源醫藥公司 | 用於恢復pah基因功能的腺相關病毒組成物及其使用方法 |
| WO2022008510A2 (en) | 2020-07-06 | 2022-01-13 | Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) | Intron-encoded extranuclear transcripts for protein translation, rna encoding, and multi-timepoint interrogation of non-coding or protein-coding rna regulation |
| EP4323515A4 (en) * | 2021-04-15 | 2025-03-12 | Altius Institute For Biomedical Sciences | Dna binding proteins for regulating gene expression |
| CA3235446A1 (en) | 2021-10-14 | 2023-04-20 | Asimov Inc. | Integrases, landing pad architectures, and engineered cells comprising the same |
| JP2024543373A (ja) | 2021-11-09 | 2024-11-21 | アシモフ,インコーポレーテッド | Aavベクター産生のための安定な産生系 |
| CA3256767A1 (en) | 2022-05-23 | 2023-11-30 | Logicbio Therapeutics, Inc. | Gene therapy compositions and their methods of use |
| WO2024064744A2 (en) * | 2022-09-20 | 2024-03-28 | Dna Twopointo, Inc. | Engineered split dhfr-based methods and systems for selecting cells that have stably acquired a heterologous polynucleotide |
| WO2024223931A1 (en) | 2023-04-28 | 2024-10-31 | AstraZeneca Ireland Limited | Manufacturing and use of recombinant self-complementary aav vectors |
Family Cites Families (31)
| Publication number | Priority date | Publication date | Assignee | Title |
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| WO1991006667A1 (en) * | 1989-11-06 | 1991-05-16 | Cell Genesys, Inc. | Production of proteins using homologous recombination |
| US5222982A (en) | 1991-02-11 | 1993-06-29 | Ommaya Ayub K | Spinal fluid driven artificial organ |
| JPH06505186A (ja) | 1991-02-11 | 1994-06-16 | オマーヤ,アユブ ケー. | 脊髄液駆動式人工器官 |
| ATE262912T1 (de) * | 1993-01-29 | 2004-04-15 | Neurospheres Holdings Ltd | Genetische modifikation von neuralen stammzellen |
| JP4375759B2 (ja) * | 1993-04-21 | 2009-12-02 | ザ・ユニバーシティ・コート・オブ・ザ・ユニバーシティ・オブ・エディンバラ | 標的とされた発現特性に従う異種遺伝子の発現 |
| US20040208846A1 (en) * | 1996-05-31 | 2004-10-21 | Wei-Wei Zhang | Mini-Ad vector for immunization |
| GB9710809D0 (en) | 1997-05-23 | 1997-07-23 | Medical Res Council | Nucleic acid binding proteins |
| GB9710807D0 (en) | 1997-05-23 | 1997-07-23 | Medical Res Council | Nucleic acid binding proteins |
| NZ504511A (en) | 1997-11-18 | 2002-12-20 | Pioneer Hi Bred Int | Recombinant proteins comprising fused first and second distinct site specific recombinase and use in methods for the integration of foreign DNA into eukaryotic genomes |
| US5929301A (en) | 1997-11-18 | 1999-07-27 | Pioneer Hi-Bred International | Nucleic acid sequence encoding FLP recombinase |
| US6140081A (en) | 1998-10-16 | 2000-10-31 | The Scripps Research Institute | Zinc finger binding domains for GNN |
| US6534261B1 (en) | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| US6453242B1 (en) | 1999-01-12 | 2002-09-17 | Sangamo Biosciences, Inc. | Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites |
| JP2002060786A (ja) | 2000-08-23 | 2002-02-26 | Kao Corp | 硬質表面用殺菌防汚剤 |
| AT5356U1 (de) | 2001-07-16 | 2002-06-25 | Uv & P Umweltman Verfahrenstec | Verfahren zur immobilisierung von verbrennungsrückständen |
| AUPR666501A0 (en) * | 2001-07-27 | 2001-08-23 | Murdoch Childrens Research Institute, The | A method for selection and agents useful for same |
| AU2002336373A1 (en) | 2001-08-20 | 2003-03-03 | The Scripps Research Institute | Zinc finger binding domains for cnn |
| US7888121B2 (en) | 2003-08-08 | 2011-02-15 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| US7972854B2 (en) | 2004-02-05 | 2011-07-05 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| CA2615532C (en) * | 2005-07-26 | 2016-06-28 | Sangamo Biosciences, Inc. | Targeted integration and expression of exogenous nucleic acid sequences |
| SI2019683T2 (sl) | 2006-04-25 | 2022-10-28 | The Regents Of The University Of California | Dajanje rastnih faktorjev za zdravljenje motenj CŽS |
| WO2008060360A2 (en) | 2006-09-28 | 2008-05-22 | Surmodics, Inc. | Implantable medical device with apertures for delivery of bioactive agents |
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| US9314005B2 (en) * | 2009-07-01 | 2016-04-19 | Transposagen Biopharmaceuticals, Inc. | Genetically modified rat models for severe combined immunodeficiency (SCID) |
| DK2462230T3 (da) * | 2009-08-03 | 2015-10-19 | Recombinetics Inc | Fremgangsmåder og sammensætninger til målrettet genmodifikation |
| DK2816112T3 (da) | 2009-12-10 | 2018-11-19 | Univ Minnesota | TAL-effektor-medieret DNA-modifikation |
| EP2660318A1 (en) * | 2010-02-09 | 2013-11-06 | Sangamo BioSciences, Inc. | Targeted genomic modification with partially single-stranded donor molecules |
| WO2011130343A1 (en) * | 2010-04-13 | 2011-10-20 | Sigma-Aldrich Co. | Biosensors comprising protein-binding domains and fluorescent proteins |
| US20140127814A1 (en) * | 2010-08-10 | 2014-05-08 | Srinivasan Chandrasegaran | Generation and use of pluripotent stem cells |
| EP2694089B1 (en) * | 2011-04-05 | 2024-06-05 | Cellectis | New tale-protein scaffolds and uses thereof |
-
2013
- 2013-03-15 WO PCT/US2013/032443 patent/WO2013158309A2/en not_active Ceased
- 2013-03-15 EP EP13778431.0A patent/EP2839013B1/en active Active
- 2013-03-15 ES ES13778431T patent/ES2828663T3/es active Active
- 2013-03-15 DK DK13778431.0T patent/DK2839013T3/da active
- 2013-03-15 EP EP20191981.8A patent/EP3839050A3/en not_active Withdrawn
- 2013-03-15 US US13/838,927 patent/US20130280222A1/en not_active Abandoned
-
2017
- 2017-03-13 US US15/457,923 patent/US20170233765A1/en not_active Abandoned
-
2020
- 2020-04-07 US US16/842,440 patent/US20210214752A1/en not_active Abandoned
-
2021
- 2021-08-26 US US17/458,044 patent/US20220204995A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| EP2839013A4 (en) | 2016-05-11 |
| EP3839050A3 (en) | 2021-09-29 |
| US20130280222A1 (en) | 2013-10-24 |
| WO2013158309A2 (en) | 2013-10-24 |
| EP2839013A2 (en) | 2015-02-25 |
| US20170233765A1 (en) | 2017-08-17 |
| US20220204995A1 (en) | 2022-06-30 |
| WO2013158309A3 (en) | 2014-01-03 |
| US20210214752A1 (en) | 2021-07-15 |
| ES2828663T3 (es) | 2021-05-27 |
| EP2839013B1 (en) | 2020-08-26 |
| EP3839050A2 (en) | 2021-06-23 |
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