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)
- Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
- Pyridine Compounds (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (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 (40)
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| KR20150027756A (ko) * | 2012-05-30 | 2015-03-12 | 베일러 칼리지 오브 메디신 | Dna 수복, 변경 및 대체를 위한 도구로서의 초나선 미니벡터 |
| ES2697912T3 (es) | 2012-07-11 | 2019-01-29 | Sangamo Therapeutics Inc | Métodos y composiciones para el tratamiento de enfermedades monogénicas |
| US10648001B2 (en) | 2012-07-11 | 2020-05-12 | Sangamo Therapeutics, Inc. | Method of treating mucopolysaccharidosis type I or II |
| US20160264995A1 (en) * | 2013-11-06 | 2016-09-15 | Hiroshima University | Vector for Nucleic Acid Insertion |
| CA2939847C (en) * | 2014-03-21 | 2023-09-05 | The Board Of Trustees Of The Leland Stanford Junior University | Genome editing without nucleases |
| SG11201608403TA (en) * | 2014-04-28 | 2016-11-29 | 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 |
| CA2967060A1 (en) * | 2014-11-10 | 2016-05-19 | Murdoch Childrens Research Institute | Vectors and methods for targeted integration in loci comprising constitutively expressed genes |
| US20170369848A1 (en) * | 2014-11-11 | 2017-12-28 | Q Therapeutics, Inc. | Engineering mesenchymal stem cells using homologous recombination |
| 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 |
| US10626372B1 (en) | 2015-01-26 | 2020-04-21 | Fate Therapeutics, Inc. | Methods and compositions for inducing hematopoietic cell differentiation |
| KR20170137730A (ko) | 2015-03-02 | 2017-12-13 | 애드베룸 바이오테크놀로지스, 인코포레이티드 | 망막 추상체에 폴리뉴클레오타이드의 유리체 내 전달을 위한 조성물 및 방법 |
| KR20170141217A (ko) * | 2015-05-12 | 2017-12-22 | 상가모 테라퓨틱스, 인코포레이티드 | 유전자 발현의 뉴클레아제-매개된 조절 |
| 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 |
| CN108473961B (zh) | 2015-11-04 | 2022-11-29 | 菲特治疗公司 | 用于诱导造血细胞分化的方法和组合物 |
| KR20250141836A (ko) | 2015-11-04 | 2025-09-29 | 페이트 세러퓨틱스, 인코포레이티드 | 만능 세포의 유전자 조작 |
| AU2016361350B2 (en) * | 2015-11-23 | 2023-04-06 | Sangamo Therapeutics, 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 |
| CA3020923A1 (en) | 2016-04-15 | 2017-10-19 | 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 |
| 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 |
| MA52431A (fr) * | 2018-03-02 | 2021-01-06 | Generation Bio Co | Identification et caractérisation de zones de sécurité du génome (gsh) chez les êtres humains et les génomes murins, et compositions de vecteurs viraux et non viraux pour une intégration ciblée au niveau d'un locus gsh identifié |
| CA3109114A1 (en) | 2018-08-10 | 2020-02-13 | Logicbio Therapeutics, Inc. | Non-disruptive gene therapy for the treatment of methaylmalonic aciduria |
| GB2611929B (en) | 2018-10-16 | 2023-11-22 | Blueallele Corp | Methods for targeted insertion of DNA in genes |
| BR112021007301A2 (pt) | 2018-10-18 | 2021-07-27 | Intellia Therapeutics, Inc. | composições e métodos para expressar fator ix |
| US20200140893A1 (en) * | 2018-11-01 | 2020-05-07 | Blueallele, Llc | 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 |
| CA3141159A1 (en) * | 2019-05-21 | 2020-11-26 | Sangamo Therapeutics, Inc. | Controlled transgene expression in regulatory t cells |
| SG11202111256XA (en) | 2019-06-07 | 2021-11-29 | Regeneron Pharma | Non-human animals comprising a humanized albumin locus |
| EP4031663A4 (en) * | 2019-09-17 | 2023-12-27 | Memorial Sloan-Kettering Cancer Center | Methods for identifying genomic safe harbors |
| PH12022553003A1 (en) * | 2020-05-04 | 2023-04-24 | Bluerock Therapeutics Lp | 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 |
| US20230250416A1 (en) | 2020-07-06 | 2023-08-10 | 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 |
| US20240218025A1 (en) * | 2021-04-15 | 2024-07-04 | Jennifer M. Cherone | DNA Binding Proteins for Regulating Gene Expression |
| AU2022366969A1 (en) | 2021-10-14 | 2024-05-09 | Asimov Inc. | Integrases, landing pad architectures, and engineered cells comprising the same |
| EP4430170A2 (en) | 2021-11-09 | 2024-09-18 | Asimov, Inc. | Stable production systems for aav vector production |
| CN119630433A (zh) | 2022-05-23 | 2025-03-14 | 逻辑生物治疗公司 | 基因疗法组合物及其使用方法 |
| 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 |
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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 |
| CA2103705C (en) | 1991-02-11 | 1996-01-30 | Ayub K. Ommaya | Spinal fluid driven artificial organ |
| DE69433661T2 (de) * | 1993-01-29 | 2005-02-10 | Neurospheres Holding Ltd., Calgary | Genetische modifikation von neuralen stammzellen |
| DK0695361T4 (da) * | 1993-04-21 | 2010-03-22 | Univ Edinburgh | Ekspression af heterologe gener ifølge en målekspressionsprofil |
| 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 |
| BR9814669A (pt) | 1997-11-18 | 2001-11-20 | Pioneer Hi Bred Int | Método para a integração de dna estranho emgenomas eucarióticos |
| 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 |
| 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 |
| US6534261B1 (en) | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| 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 |
| 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 |
| WO2007014275A2 (en) * | 2005-07-26 | 2007-02-01 | 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 |
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| US8110379B2 (en) * | 2007-04-26 | 2012-02-07 | Sangamo Biosciences, Inc. | Targeted integration into the PPP1R12C locus |
| EP2257250A2 (en) | 2008-01-29 | 2010-12-08 | Gilbert H. Kliman | Drug delivery devices, kits and methods therefor |
| WO2011002988A1 (en) * | 2009-07-01 | 2011-01-06 | Transposagen Biopharmaceuticals, Inc. | Genetically modified rat models for severe combined immunodeficiency (scid) |
| NZ598457A (en) * | 2009-08-03 | 2014-06-27 | Recombinetics Inc | Methods and compositions for targeted gene modification |
| CA2783351C (en) | 2009-12-10 | 2021-09-07 | Regents Of The University Of Minnesota | Tal effector-mediated dna modification |
| WO2011100058A1 (en) * | 2010-02-09 | 2011-08-18 | Sangamo Biosciences, Inc. | Targeted genomic modification with partially single-stranded donor molecules |
| EP2558574B1 (en) * | 2010-04-13 | 2015-03-18 | Sigma-Aldrich Co. LLC | Use of endogenous promoters to express heterologous proteins |
| US20140127814A1 (en) * | 2010-08-10 | 2014-05-08 | Srinivasan Chandrasegaran | Generation and use of pluripotent stem cells |
| EP2694091B1 (en) * | 2011-04-05 | 2019-03-13 | Cellectis | Method for the generation of compact tale-nucleases and uses thereof |
-
2013
- 2013-03-15 ES ES13778431T patent/ES2828663T3/es active Active
- 2013-03-15 DK DK13778431.0T patent/DK2839013T3/da active
- 2013-03-15 US US13/838,927 patent/US20130280222A1/en not_active Abandoned
- 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 EP EP20191981.8A patent/EP3839050A3/en active Pending
-
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 active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP2839013A4 (en) | 2016-05-11 |
| EP2839013A2 (en) | 2015-02-25 |
| US20210214752A1 (en) | 2021-07-15 |
| EP3839050A2 (en) | 2021-06-23 |
| WO2013158309A2 (en) | 2013-10-24 |
| US20220204995A1 (en) | 2022-06-30 |
| EP3839050A3 (en) | 2021-09-29 |
| US20170233765A1 (en) | 2017-08-17 |
| WO2013158309A3 (en) | 2014-01-03 |
| US20130280222A1 (en) | 2013-10-24 |
| ES2828663T3 (es) | 2021-05-27 |
| EP2839013B1 (en) | 2020-08-26 |
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