PL3152312T3 - Sposoby i kompozycje do modyfikowania docelowego locus - Google Patents
Sposoby i kompozycje do modyfikowania docelowego locusInfo
- Publication number
- PL3152312T3 PL3152312T3 PL15729724T PL15729724T PL3152312T3 PL 3152312 T3 PL3152312 T3 PL 3152312T3 PL 15729724 T PL15729724 T PL 15729724T PL 15729724 T PL15729724 T PL 15729724T PL 3152312 T3 PL3152312 T3 PL 3152312T3
- Authority
- PL
- Poland
- Prior art keywords
- modifying
- compositions
- methods
- targeted locus
- locus
- Prior art date
Links
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
- C12N15/87—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
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K67/00—Rearing or breeding animals, not otherwise provided for; New breeds of animals
- A01K67/027—New breeds of vertebrates
- A01K67/0275—Genetically modified vertebrates, e.g. transgenic
- A01K67/0278—Humanized animals, e.g. knockin
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
- C07K14/70503—Immunoglobulin superfamily
- C07K14/7051—T-cell receptor (TcR)-CD3 complex
-
- 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
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing nucleic acids
-
- 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
- C12N15/64—General methods for preparing the vector, for introducing it into the cell or for selecting the vector-containing host
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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
- C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
-
- 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
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2207/00—Modified animals
- A01K2207/15—Humanized animals
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/15—Animals comprising multiple alterations of the genome, by transgenesis or homologous recombination, e.g. obtained by cross-breeding
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/035—Animal model for multifactorial diseases
- A01K2267/0387—Animal model for diseases of the immune system
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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
- C12N15/8509—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
- C12N2015/8527—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic for producing animal models, e.g. for tests or diseases
-
- 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
Applications Claiming Priority (4)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201462008832P | 2014-06-06 | 2014-06-06 | |
US201462017916P | 2014-06-27 | 2014-06-27 | |
EP15729724.3A EP3152312B1 (en) | 2014-06-06 | 2015-06-05 | Methods and compositions for modifying a targeted locus |
PCT/US2015/034503 WO2015188109A1 (en) | 2014-06-06 | 2015-06-05 | Methods and compositions for modifying a targeted locus |
Publications (1)
Publication Number | Publication Date |
---|---|
PL3152312T3 true PL3152312T3 (pl) | 2020-08-10 |
Family
ID=53404960
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PL15729724T PL3152312T3 (pl) | 2014-06-06 | 2015-06-05 | Sposoby i kompozycje do modyfikowania docelowego locus |
Country Status (24)
Country | Link |
---|---|
US (3) | US10106820B2 (pl) |
EP (2) | EP3152312B1 (pl) |
JP (2) | JP6688231B2 (pl) |
KR (1) | KR102374379B1 (pl) |
CN (2) | CN106795521B (pl) |
AU (1) | AU2015269187B2 (pl) |
BR (1) | BR112016028564A2 (pl) |
CA (1) | CA2950173C (pl) |
CY (1) | CY1122897T1 (pl) |
DK (1) | DK3152312T3 (pl) |
ES (1) | ES2784754T3 (pl) |
HR (1) | HRP20200529T1 (pl) |
HU (1) | HUE049776T2 (pl) |
IL (1) | IL249042B (pl) |
LT (1) | LT3152312T (pl) |
MX (1) | MX2016016133A (pl) |
NZ (1) | NZ727481A (pl) |
PL (1) | PL3152312T3 (pl) |
PT (1) | PT3152312T (pl) |
RS (1) | RS60359B1 (pl) |
RU (1) | RU2704283C9 (pl) |
SG (2) | SG10201913804WA (pl) |
SI (1) | SI3152312T1 (pl) |
WO (1) | WO2015188109A1 (pl) |
Families Citing this family (66)
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JP6261500B2 (ja) | 2011-07-22 | 2018-01-17 | プレジデント アンド フェローズ オブ ハーバード カレッジ | ヌクレアーゼ切断特異性の評価および改善 |
CN104364380B (zh) | 2012-04-25 | 2018-10-09 | 瑞泽恩制药公司 | 核酸酶介导的使用大靶向载体的靶向 |
SG10201706741VA (en) | 2013-02-20 | 2017-10-30 | Regeneron Pharma | Genetic modification of rats |
PT3456831T (pt) | 2013-04-16 | 2021-09-10 | Regeneron Pharma | Modificação alvejada do genoma de rato |
US9163284B2 (en) | 2013-08-09 | 2015-10-20 | President And Fellows Of Harvard College | Methods for identifying a target site of a Cas9 nuclease |
US9359599B2 (en) | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
US9340800B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | Extended DNA-sensing GRNAS |
HUE041331T2 (hu) | 2013-12-11 | 2019-05-28 | Regeneron Pharma | Módszerek és készítmények a genom célzott módosításához |
KR102170502B1 (ko) | 2013-12-11 | 2020-10-28 | 리제너론 파마슈티칼스 인코포레이티드 | 게놈의 표적화된 변형을 위한 방법 및 조성물 |
US11053481B2 (en) | 2013-12-12 | 2021-07-06 | President And Fellows Of Harvard College | Fusions of Cas9 domains and nucleic acid-editing domains |
SG10201913804WA (en) | 2014-06-06 | 2020-03-30 | Regeneron Pharma | Methods and compositions for modifying a targeted locus |
EP3161128B1 (en) | 2014-06-26 | 2018-09-26 | Regeneron Pharmaceuticals, Inc. | Methods and compositions for targeted genetic modifications and methods of use |
US10077453B2 (en) | 2014-07-30 | 2018-09-18 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
RU2706965C2 (ru) | 2014-10-15 | 2019-11-21 | Регенерон Фармасьютикалз, Инк. | Способы и композиции для получения или поддержания плюрипотентных клеток |
PT3221457T (pt) | 2014-11-21 | 2019-06-27 | Regeneron Pharma | Métodos e composições para modificação genética visada através da utilização de arn guia emparelhados |
JP6840077B2 (ja) | 2014-12-19 | 2021-03-10 | リジェネロン・ファーマシューティカルズ・インコーポレイテッドRegeneron Pharmaceuticals, Inc. | 単一ステップの複数標的化を通じた標的化された遺伝子修飾のための方法及び組成物 |
DK3680329T3 (da) | 2015-10-05 | 2021-08-23 | Prec Biosciences Inc | Modificerede meganukleaser med genkendelsesekvenser der findes i genet for den humane t-cellereceptor-alpha-konstantregion |
DK3359184T3 (da) | 2015-10-05 | 2020-06-15 | Prec Biosciences Inc | Genetisk modificerede celler, der omfatter et modificeret gen fra konstant alfaregion i human t-cellereceptor |
WO2017070632A2 (en) | 2015-10-23 | 2017-04-27 | President And Fellows Of Harvard College | Nucleobase editors and uses thereof |
JP2019509738A (ja) * | 2016-03-11 | 2019-04-11 | ブルーバード バイオ, インコーポレイテッド | ゲノム編集された免疫エフェクター細胞 |
KR102437015B1 (ko) | 2016-04-15 | 2022-08-29 | 메모리얼 슬로안 케터링 캔서 센터 | 유전자이식 t 세포 및 키메라 항원 수용체 t 세포 조성물 및 관련 방법 |
EP3457840B1 (en) * | 2016-05-20 | 2024-04-10 | Regeneron Pharmaceuticals, Inc. | Methods for breaking immunological tolerance using multiple guide rnas |
DK3462853T3 (da) | 2016-06-03 | 2023-04-03 | Regeneron Pharma | Gnavere, der udtrykker exogen, terminal deoxynukleotidyltransferase |
CN107513538A (zh) * | 2016-06-17 | 2017-12-26 | 北京大学 | 基因敲除方法 |
US20180004537A1 (en) | 2016-07-01 | 2018-01-04 | Microsoft Technology Licensing, Llc | Molecular State Machines |
US11359234B2 (en) * | 2016-07-01 | 2022-06-14 | Microsoft Technology Licensing, Llc | Barcoding sequences for identification of gene expression |
WO2018027078A1 (en) | 2016-08-03 | 2018-02-08 | President And Fellows Of Harard College | Adenosine nucleobase editors and uses thereof |
CA3033327A1 (en) | 2016-08-09 | 2018-02-15 | President And Fellows Of Harvard College | Programmable cas9-recombinase fusion proteins and uses thereof |
WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
GB2573062A (en) | 2016-10-14 | 2019-10-23 | Harvard College | AAV delivery of nucleobase editors |
WO2018128691A1 (en) | 2016-11-04 | 2018-07-12 | Regeneron Pharmaceuticals, Inc. | Non-human animals having an engineered immunoglobulin lambda light chain locus |
WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
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 |
WO2018151514A1 (ko) * | 2017-02-16 | 2018-08-23 | 고려대학교 산학협력단 | 골재생 효능이 우수한 골질환 예방 또는 치료용 조성물 |
US11898179B2 (en) | 2017-03-09 | 2024-02-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
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KR20190130613A (ko) | 2017-03-23 | 2019-11-22 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제 |
US11560566B2 (en) | 2017-05-12 | 2023-01-24 | President And Fellows Of Harvard College | Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation |
JP2020529834A (ja) | 2017-06-30 | 2020-10-15 | プレシジョン バイオサイエンシズ,インク. | T細胞受容体アルファ遺伝子の改変されたイントロンを含む遺伝子改変t細胞 |
EP3652320A4 (en) * | 2017-07-12 | 2021-04-14 | Mayo Foundation for Medical Education and Research | MATERIALS AND PROCEDURES FOR EFFICIENT TARGETED KNOCK-IN OR GENERAL REPLACEMENT |
CN111801345A (zh) | 2017-07-28 | 2020-10-20 | 哈佛大学的校长及成员们 | 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物 |
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WO2019139645A2 (en) | 2017-08-30 | 2019-07-18 | President And Fellows Of Harvard College | High efficiency base editors comprising gam |
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WO2019079347A1 (en) | 2017-10-16 | 2019-04-25 | The Broad Institute, Inc. | USES OF BASIC EDITORS ADENOSINE |
US20210017544A1 (en) * | 2017-12-22 | 2021-01-21 | Novozymes A/S | Counter-Selection by Inhibition of Conditionally Essential Genes |
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KR20200103769A (ko) * | 2018-01-23 | 2020-09-02 | 기초과학연구원 | 연장된 단일 가이드 rna 및 그 용도 |
CN111902164A (zh) * | 2018-02-01 | 2020-11-06 | 同源药物公司 | 用于恢复pah基因功能的腺相关病毒组合物及其使用方法 |
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IL271656A (en) | 2019-12-22 | 2021-06-30 | Yeda Res & Dev | System and methods for identifying cells that have undergone genome editing |
CN116096873A (zh) | 2020-05-08 | 2023-05-09 | 布罗德研究所股份有限公司 | 同时编辑靶标双链核苷酸序列的两条链的方法和组合物 |
WO2022104344A2 (en) * | 2020-11-10 | 2022-05-19 | The Board Of Trustees Of The Leland Stanford Junior University | Knock-in of large dna for long-term high genomic expression |
CN114763559A (zh) * | 2021-01-15 | 2022-07-19 | 中国农业大学 | 一种不依赖于同源重组的靶向性基因捕获系统及其应用 |
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