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)
- Wood Science & Technology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Cell Biology (AREA)
- Mycology (AREA)
- Medicinal Chemistry (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (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)
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) |
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| CA2876860A1 (en) * | 2012-05-30 | 2013-12-05 | Baylor College Of Medicine | Supercoiled minivectors as a tool for dna repair, alteration and replacement |
| EP3196301B1 (en) * | 2012-07-11 | 2018-10-17 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of monogenic diseases |
| US10648001B2 (en) | 2012-07-11 | 2020-05-12 | Sangamo Therapeutics, Inc. | Method of treating mucopolysaccharidosis type I or II |
| JP5900942B2 (ja) * | 2013-11-06 | 2016-04-06 | 国立大学法人広島大学 | 核酸挿入用ベクター |
| BR112016019940A2 (pt) * | 2014-03-21 | 2017-10-24 | Univ Leland Stanford Junior | edição de genoma sem nucleases |
| EP3137633A4 (en) * | 2014-04-28 | 2017-11-29 | Sigma-Aldrich Co. LLC | Epigenetic modification of mammalian genomes using targeted endonucleases |
| WO2016081032A2 (en) * | 2014-07-11 | 2016-05-26 | The Texas A&M University System | Mesenchymal stem cells derived from induced pluripotent stem cells |
| EP3218503A4 (en) * | 2014-11-10 | 2018-06-06 | Murdoch Childrens Research Institute | Vectors and methods for targeted integration in loci comprising constitutively expressed genes |
| CN107532142A (zh) * | 2014-11-11 | 2018-01-02 | 应用干细胞有限公司 | 使用同源重组改造间充质干细胞 |
| JP6814155B2 (ja) * | 2014-12-12 | 2021-01-13 | ジュー，ジェイムズ | 対象とする細胞を選択的に除去する方法及び組成物 |
| WO2016123100A1 (en) | 2015-01-26 | 2016-08-04 | Fate Therapeutics, Inc. | Methods and compositions for inducing hematopoietic cell differentiation |
| US11021519B2 (en) | 2015-03-02 | 2021-06-01 | Adverum Biotechnologies, Inc. | Compositions and methods for intravitreal delivery of polynucleotides to retinal cones |
| AU2016261927B2 (en) * | 2015-05-12 | 2022-04-07 | 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 |
| KR20250141836A (ko) | 2015-11-04 | 2025-09-29 | 페이트 세러퓨틱스, 인코포레이티드 | 만능 세포의 유전자 조작 |
| SG11201803145RA (en) | 2015-11-04 | 2018-05-30 | Fate Therapeutics Inc | Methods and compositions for inducing hematopoietic cell differentiation |
| US10639383B2 (en) * | 2015-11-23 | 2020-05-05 | Sangamo Therapeutics, Inc. | Methods and compositions for engineering immunity |
| AU2017250683A1 (en) * | 2016-04-14 | 2018-11-01 | Boco Silicon Valley, Inc. | Genome editing of human neural stem cells using nucleases |
| EP4628587A3 (en) | 2016-04-15 | 2026-02-25 | Memorial Sloan Kettering Cancer Center | Transgenic t cell and chimeric antigen receptor t cell compositions and related methods |
| 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 |
| CN111344403B (zh) * | 2017-09-15 | 2025-05-06 | 利兰斯坦福初级大学董事会 | 遗传工程细胞的多元性产生和条形码编制 |
| US20190256867A1 (en) | 2018-02-01 | 2019-08-22 | Homology Medicines, Inc. | Adeno-associated virus compositions for restoring pah gene function and methods of use thereof |
| EP3759226A4 (en) * | 2018-03-02 | 2022-06-15 | Generation Bio Co. | IDENTIFICATION AND CHARACTERIZATION OF GENOMIC SAFE HARBOR (GSH) IN HUMAN AND MURINE GENOMES AND VIRAL AND NON-VIRAL VECTOR COMPOSITIONS FOR TARGETED INTEGRATION AT IDENTIFIED GSH LOCI |
| MA53252A (fr) | 2018-08-10 | 2021-09-15 | Logicbio Therapeutics Inc | Thérapie génique non perturbatrice pour le traitement d'un mma |
| SG11202103917VA (en) | 2018-10-16 | 2021-05-28 | Blueallele Llc | Methods for targeted insertion of dna in genes |
| AU2019360270B2 (en) | 2018-10-18 | 2025-08-07 | Intellia Therapeutics, Inc. | Compositions and methods for expressing factor IX. |
| EP3874046A2 (en) * | 2018-11-01 | 2021-09-08 | BlueAllele Corporation | Methods for altering gene expression for genetic disorders |
| 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 |
| JP2022533713A (ja) * | 2019-05-21 | 2022-07-25 | サンガモ セラピューティクス， インコーポレイテッド | 調節性ｔ細胞における制御された導入遺伝子発現 |
| MX2021015122A (es) | 2019-06-07 | 2022-04-06 | Regeneron Pharma | Animales no humanos que comprenden un locus de albumina humanizado. |
| WO2021055592A1 (en) * | 2019-09-17 | 2021-03-25 | Memorial Sloan-Kettering Cancer Center | Methods for identifying genomic safe harbors |
| US12521451B2 (en) | 2019-11-08 | 2026-01-13 | Regeneron Pharmaceuticals, Inc. | CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy |
| JP2023524976A (ja) * | 2020-05-04 | 2023-06-14 | エディタス・メディシン、インコーポレイテッド | 必須遺伝子ノックインによる選択 |
| 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 |
| WO2022221694A1 (en) * | 2021-04-15 | 2022-10-20 | Altius Institute For Biomedical Sciences | Dna binding proteins for regulating gene expression |
| JP2024535618A (ja) | 2021-10-14 | 2024-09-30 | アシモフ，インコーポレーテッド | インテグラーゼ、ランディングパッド構造、およびそれを含む操作された細胞 |
| IL312487A (en) | 2021-11-09 | 2024-07-01 | Asimov Inc | Stable production systems for adeno-associated virus vector production |
| WO2023230098A1 (en) | 2022-05-23 | 2023-11-30 | Logicbio Therapeutics, Inc. | Gene therapy compositions and methods of use thereof |
| US20250207109A1 (en) * | 2022-09-20 | 2025-06-26 | 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 |
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| EP0945515A3 (en) * | 1989-11-06 | 2002-08-21 | 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 |
| FI953569A7 (fi) * | 1993-01-29 | 1995-09-25 | Neurospheres Ltd | Neuraalisten kantasolujen geneettinen modifikaatio |
| 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 |
| GB9710807D0 (en) | 1997-05-23 | 1997-07-23 | Medical Res Council | Nucleic acid binding proteins |
| GB9710809D0 (en) | 1997-05-23 | 1997-07-23 | Medical Res Council | Nucleic acid binding proteins |
| ES2256972T3 (es) | 1997-11-18 | 2006-07-16 | Pioneer Hi-Bred International, Inc. | Metodo novedoso para la integracion de un adn foraneo dentro de genomas de eucariotas. |
| 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 |
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| 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 |
| JP2005500061A (ja) | 2001-08-20 | 2005-01-06 | ザ スクリップス リサーチ インスティテュート | Ｃｎｎについての亜鉛フィンガー結合ドメイン |
| US20070134796A1 (en) * | 2005-07-26 | 2007-06-14 | Sangamo Biosciences, Inc. | Targeted integration and expression of exogenous nucleic acid sequences |
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| DK2019683T4 (da) | 2006-04-25 | 2022-08-29 | Univ California | Indgivelse af vækstfaktorer til behandling af CNS-lidelser |
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| DE602008003684D1 (de) * | 2007-04-26 | 2011-01-05 | Sangamo Biosciences Inc | Gezielte integration in die ppp1r12c-position |
| WO2009097468A2 (en) | 2008-01-29 | 2009-08-06 | Kliman Gilbert H | Drug delivery devices, kits and methods therefor |
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| WO2011017315A2 (en) * | 2009-08-03 | 2011-02-10 | Recombinetics, Inc. | Methods and compositions for targeted gene modification |
| WO2011072246A2 (en) | 2009-12-10 | 2011-06-16 | Regents Of The University Of Minnesota | Tal effector-mediated dna modification |
| 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 |
| CA3111953C (en) * | 2011-04-05 | 2023-10-24 | Cellectis | Method for the generation of compact tale-nucleases and uses thereof |
-
2013
- 2013-03-15 EP EP13778431.0A patent/EP2839013B1/en active Active
- 2013-03-15 WO PCT/US2013/032443 patent/WO2013158309A2/en not_active Ceased
- 2013-03-15 US US13/838,927 patent/US20130280222A1/en not_active Abandoned
- 2013-03-15 EP EP20191981.8A patent/EP3839050A3/en not_active Withdrawn
- 2013-03-15 DK DK13778431.0T patent/DK2839013T3/da active
- 2013-03-15 ES ES13778431T patent/ES2828663T3/es active Active
-
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 |
|---|---|
| WO2013158309A2 (en) | 2013-10-24 |
| US20130280222A1 (en) | 2013-10-24 |
| WO2013158309A3 (en) | 2014-01-03 |
| US20210214752A1 (en) | 2021-07-15 |
| EP2839013B1 (en) | 2020-08-26 |
| US20170233765A1 (en) | 2017-08-17 |
| EP2839013A2 (en) | 2015-02-25 |
| EP3839050A3 (en) | 2021-09-29 |
| US20220204995A1 (en) | 2022-06-30 |
| EP3839050A2 (en) | 2021-06-23 |
| EP2839013A4 (en) | 2016-05-11 |
| ES2828663T3 (es) | 2021-05-27 |
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