JP2019511243A - インビボのデュアルリコンビナーゼ媒介性カセット交換(dRMCE)のためのシステム及び方法ならびにその疾患モデル - Google Patents
インビボのデュアルリコンビナーゼ媒介性カセット交換(dRMCE)のためのシステム及び方法ならびにその疾患モデル Download PDFInfo
- Publication number
- JP2019511243A JP2019511243A JP2018557283A JP2018557283A JP2019511243A JP 2019511243 A JP2019511243 A JP 2019511243A JP 2018557283 A JP2018557283 A JP 2018557283A JP 2018557283 A JP2018557283 A JP 2018557283A JP 2019511243 A JP2019511243 A JP 2019511243A
- Authority
- JP
- Japan
- Prior art keywords
- cells
- rna
- recombinase
- transgene
- various embodiments
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
Images
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
- C12N15/63—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
-
- 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
- A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
- A01K67/027—New or modified breeds of vertebrates
- A01K67/0275—Genetically modified vertebrates, e.g. transgenic
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K49/00—Preparations for testing in vivo
- A61K49/0004—Screening or testing of compounds for diagnosis of disorders, assessment of conditions, e.g. renal clearance, gastric emptying, testing for diabetes, allergy, rheuma, pancreas functions
- A61K49/0008—Screening agents using (non-human) animal models or transgenic animal models or chimeric hosts, e.g. Alzheimer disease animal model, transgenic model for heart failure
-
- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
-
- 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
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
-
- 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
- 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; AVICULTURE; APICULTURE; PISCICULTURE; 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
- A01K2267/0331—Animal model for proliferative 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
- 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
- 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
- C12N2015/8572—Animal models for proliferative diseases, e.g. comprising an oncogene
-
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/30—Vector systems comprising sequences for excision in presence of a recombinase, e.g. loxP or FRT
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
Landscapes
- Life Sciences & Earth Sciences (AREA)
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Zoology (AREA)
- Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Veterinary Medicine (AREA)
- Biochemistry (AREA)
- Environmental Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Biodiversity & Conservation Biology (AREA)
- Animal Husbandry (AREA)
- Epidemiology (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Toxicology (AREA)
- Urology & Nephrology (AREA)
- Diabetes (AREA)
- Rheumatology (AREA)
- Pharmacology & Pharmacy (AREA)
- Pathology (AREA)
- Gastroenterology & Hepatology (AREA)
- Endocrinology (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)
- Investigating Or Analysing Biological Materials (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022036725A JP2022091801A (ja) | 2016-01-26 | 2022-03-10 | インビボのデュアルリコンビナーゼ媒介性カセット交換(dRMCE)のためのシステム及び方法ならびにその疾患モデル |
| JP2024025071A JP2024063065A (ja) | 2016-01-26 | 2024-02-22 | インビボのデュアルリコンビナーゼ媒介性カセット交換(dRMCE)のためのシステム及び方法ならびにその疾患モデル |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662287197P | 2016-01-26 | 2016-01-26 | |
| US62/287,197 | 2016-01-26 | ||
| PCT/US2016/069442 WO2017131926A1 (en) | 2016-01-26 | 2016-12-30 | SYSTEMS AND METHODS FOR IN VIVO DUAL RECOMBINASE-MEDIATED CASSETTE EXCHANGE (dRMCE) AND DISEASE MODELS THEREOF |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022036725A Division JP2022091801A (ja) | 2016-01-26 | 2022-03-10 | インビボのデュアルリコンビナーゼ媒介性カセット交換(dRMCE)のためのシステム及び方法ならびにその疾患モデル |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2019511243A true JP2019511243A (ja) | 2019-04-25 |
| JP2019511243A5 JP2019511243A5 (https=) | 2020-02-06 |
Family
ID=59398838
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018557283A Pending JP2019511243A (ja) | 2016-01-26 | 2016-12-30 | インビボのデュアルリコンビナーゼ媒介性カセット交換(dRMCE)のためのシステム及び方法ならびにその疾患モデル |
| JP2022036725A Pending JP2022091801A (ja) | 2016-01-26 | 2022-03-10 | インビボのデュアルリコンビナーゼ媒介性カセット交換(dRMCE)のためのシステム及び方法ならびにその疾患モデル |
| JP2024025071A Pending JP2024063065A (ja) | 2016-01-26 | 2024-02-22 | インビボのデュアルリコンビナーゼ媒介性カセット交換(dRMCE)のためのシステム及び方法ならびにその疾患モデル |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022036725A Pending JP2022091801A (ja) | 2016-01-26 | 2022-03-10 | インビボのデュアルリコンビナーゼ媒介性カセット交換(dRMCE)のためのシステム及び方法ならびにその疾患モデル |
| JP2024025071A Pending JP2024063065A (ja) | 2016-01-26 | 2024-02-22 | インビボのデュアルリコンビナーゼ媒介性カセット交換(dRMCE)のためのシステム及び方法ならびにその疾患モデル |
Country Status (8)
| Country | Link |
|---|---|
| US (2) | US11466290B2 (https=) |
| EP (1) | EP3408396A4 (https=) |
| JP (3) | JP2019511243A (https=) |
| KR (1) | KR102811061B1 (https=) |
| CN (1) | CN108884472A (https=) |
| AU (1) | AU2016389495B2 (https=) |
| CA (1) | CA3012042A1 (https=) |
| WO (1) | WO2017131926A1 (https=) |
Cited By (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2022536934A (ja) * | 2019-06-17 | 2022-08-22 | シーダーズ-サイナイ メディカル センター | in vivo二重リコンビナーゼ媒介カセット交換(dRMCE)のためのシステム及び方法ならびにその疾患モデル |
| US11466290B2 (en) | 2016-01-26 | 2022-10-11 | Cedars-Sinai Medical Center | Systems and methods for in vivo dual recombinase-mediated cassette exchange (dRMCE) and disease models thereof |
| US12258571B2 (en) | 2018-03-16 | 2025-03-25 | Cedars-Sinai Medical Center | Methods and compositions for inducible expression of neurotrophic factors |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN109321599B (zh) * | 2018-09-27 | 2023-08-22 | 同济大学 | 人多能干细胞中的谱系示踪系统的构建与应用 |
| CN109943564B (zh) * | 2019-03-28 | 2022-03-15 | 上海市中医老年医学研究所 | Amh基因定点敲入2A-Cre的杂合子小鼠模型构建方法及其应用 |
| CN118547004A (zh) * | 2019-06-19 | 2024-08-27 | 豪夫迈·罗氏有限公司 | 通过使用Cre mRNA进行的靶向整合来产生蛋白质表达细胞的方法 |
| US20250354186A1 (en) | 2022-06-06 | 2025-11-20 | Kyowa Hakko Bio Co., Ltd. | Microorganism having improved 3'-sialyllactose productivity and production method of 3'-sialyllactose |
Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2011001247A2 (en) * | 2009-06-29 | 2011-01-06 | University Of Basel | Methods and kits for high efficiency engineering of conditional mouse alleles |
| WO2014204724A1 (en) * | 2013-06-17 | 2014-12-24 | The Broad Institute Inc. | Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation |
Family Cites Families (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7575924B2 (en) * | 2000-11-13 | 2009-08-18 | Research Development Foundation | Methods and compositions relating to improved lentiviral vectors and their applications |
| WO2003020743A1 (en) * | 2001-09-05 | 2003-03-13 | Regeneron Pharmaceuticals, Inc. | Methods of expressing transgenes |
| CA2484223A1 (en) | 2002-04-25 | 2003-11-06 | Wisconsin Alumni Research Foundation | Use of human neural stem cells secreting gdnf for treatment of parkinson's and other neurodegenerative diseases |
| GB2404382B (en) | 2003-07-28 | 2008-01-30 | Oxitec Ltd | Pest control |
| WO2008079608A1 (en) * | 2006-12-21 | 2008-07-03 | Genentech, Inc. | Compositions and methods for the expression of nucleic acids |
| US8592211B2 (en) | 2009-03-20 | 2013-11-26 | The Rockefeller University | Enhanced PiggyBac transposon and methods for transposon mutagenesis |
| US9528124B2 (en) * | 2013-08-27 | 2016-12-27 | Recombinetics, Inc. | Efficient non-meiotic allele introgression |
| WO2013155222A2 (en) | 2012-04-10 | 2013-10-17 | The Regents Of The University Of California | Brain-specific enhancers for cell-based therapy |
| WO2014127289A1 (en) | 2013-02-15 | 2014-08-21 | International Stem Cell Corporation | Use of neural cells derived from human pluripotent stem cells for the treatment of neurodegenerative diseases |
| JP6473077B2 (ja) | 2013-03-21 | 2019-02-20 | 国立大学法人京都大学 | 神経分化誘導用の多能性幹細胞 |
| ES2939807T3 (es) | 2014-03-21 | 2023-04-27 | Fujifilm Cellular Dynamics Inc | Producción de neuronas dopaminérgicas del mesencéfalo y métodos para su utilización |
| AU2015234242B2 (en) | 2014-03-21 | 2020-09-24 | Children's Medical Research Institute | Stable gene transfer to proliferating cells |
| US20170274048A1 (en) | 2014-03-28 | 2017-09-28 | Buck Institute For Research On Aging | Methods and compositions for modulating the imune system |
| EP3584312A1 (en) | 2014-06-27 | 2019-12-25 | Angiocrine Bioscience, Inc. | Neural cells expressing adenovirus e4orf1, and methods of making and using the same |
| KR20250141836A (ko) | 2015-11-04 | 2025-09-29 | 페이트 세러퓨틱스, 인코포레이티드 | 만능 세포의 유전자 조작 |
| JP2019511243A (ja) | 2016-01-26 | 2019-04-25 | シーダーズ−サイナイ メディカル センター | インビボのデュアルリコンビナーゼ媒介性カセット交換(dRMCE)のためのシステム及び方法ならびにその疾患モデル |
| US12258571B2 (en) | 2018-03-16 | 2025-03-25 | Cedars-Sinai Medical Center | Methods and compositions for inducible expression of neurotrophic factors |
| CA3143981A1 (en) | 2019-06-17 | 2020-12-24 | Cedars-Sinai Medical Center | Systems and methods for in vivo dual recombinase-mediated cassette exchange (drmce) and disease models thereof |
-
2016
- 2016-12-30 JP JP2018557283A patent/JP2019511243A/ja active Pending
- 2016-12-30 WO PCT/US2016/069442 patent/WO2017131926A1/en not_active Ceased
- 2016-12-30 CN CN201680084065.6A patent/CN108884472A/zh active Pending
- 2016-12-30 CA CA3012042A patent/CA3012042A1/en active Pending
- 2016-12-30 AU AU2016389495A patent/AU2016389495B2/en active Active
- 2016-12-30 US US16/071,407 patent/US11466290B2/en active Active
- 2016-12-30 EP EP16888589.5A patent/EP3408396A4/en active Pending
- 2016-12-30 KR KR1020187023347A patent/KR102811061B1/ko active Active
-
2022
- 2022-03-10 JP JP2022036725A patent/JP2022091801A/ja active Pending
- 2022-09-08 US US17/930,558 patent/US20230084201A1/en active Pending
-
2024
- 2024-02-22 JP JP2024025071A patent/JP2024063065A/ja active Pending
Patent Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2011001247A2 (en) * | 2009-06-29 | 2011-01-06 | University Of Basel | Methods and kits for high efficiency engineering of conditional mouse alleles |
| WO2014204724A1 (en) * | 2013-06-17 | 2014-12-24 | The Broad Institute Inc. | Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation |
Non-Patent Citations (3)
| Title |
|---|
| BIOTECHNIQUES (2013) VOL.55, NO.5, PP.233-242, JPN6020046795, ISSN: 0004637484 * |
| NATURE METHODS (2010) VOL.7, NO.11, PP.893-895, ONLINE METHODS, JPN6020046794, ISSN: 0004400970 * |
| PATHOGENETICS (2008) VOL.1, NO.3, PP.1-10, JPN6020046793, ISSN: 0004637485 * |
Cited By (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11466290B2 (en) | 2016-01-26 | 2022-10-11 | Cedars-Sinai Medical Center | Systems and methods for in vivo dual recombinase-mediated cassette exchange (dRMCE) and disease models thereof |
| US12258571B2 (en) | 2018-03-16 | 2025-03-25 | Cedars-Sinai Medical Center | Methods and compositions for inducible expression of neurotrophic factors |
| JP2022536934A (ja) * | 2019-06-17 | 2022-08-22 | シーダーズ-サイナイ メディカル センター | in vivo二重リコンビナーゼ媒介カセット交換(dRMCE)のためのシステム及び方法ならびにその疾患モデル |
Also Published As
| Publication number | Publication date |
|---|---|
| EP3408396A1 (en) | 2018-12-05 |
| KR20180101535A (ko) | 2018-09-12 |
| US20230084201A1 (en) | 2023-03-16 |
| AU2016389495A1 (en) | 2018-08-16 |
| WO2017131926A1 (en) | 2017-08-03 |
| KR102811061B1 (ko) | 2025-05-21 |
| EP3408396A4 (en) | 2019-10-09 |
| CA3012042A1 (en) | 2017-08-03 |
| CN108884472A (zh) | 2018-11-23 |
| US11466290B2 (en) | 2022-10-11 |
| JP2022091801A (ja) | 2022-06-21 |
| JP2024063065A (ja) | 2024-05-10 |
| US20190390222A1 (en) | 2019-12-26 |
| AU2016389495B2 (en) | 2023-03-30 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US20230084201A1 (en) | SYSTEMS AND METHODS FOR IN VIVO DUAL RECOMBINASE-MEDIATED CASSETTE EXCHANGE (dRMCE) AND DISEASE MODELS THEREOF | |
| US20160186208A1 (en) | Methods of Mutating, Modifying or Modulating Nucleic Acid in a Cell or Nonhuman Mammal | |
| US20220304286A1 (en) | SYSTEMS AND METHODS FOR IN VIVO DUAL RECOMBINASE-MEDIATED CASSETTE EXCHANGE (dRMCE) AND DISEASE MODELS THEREOF | |
| JP2019528068A (ja) | Dnaメチル化の編集方法 | |
| Bodea et al. | LINE-1 retrotransposons contribute to mouse PV interneuron development | |
| Aparisi Rey et al. | Gadd45α modulates aversive learning through post‐transcriptional regulation of memory‐related mRNAs | |
| JP7641033B2 (ja) | ヒト組織幹細胞及びその使用 | |
| Imle et al. | Somatic gene delivery faithfully recapitulates a molecular spectrum of high-risk sarcomas | |
| Zhang et al. | Genome writing to dissect consequences of SVA retrotransposon disease X-Linked Dystonia Parkinsonism | |
| Tennant et al. | Fluorescent in vivo editing reporter (FIVER): a novel multispectral reporter of in vivo genome editing | |
| US11174495B2 (en) | Reporter system for detecting and targeting activated cells | |
| WO2012004346A1 (en) | Genetically engineered stem cells, method of production and uses thereof | |
| US10612022B2 (en) | ETS factors regulate neural stem cell depletion and gliogenesis in Ras pathway glioma | |
| Kim et al. | Generating in vivo somatic mouse mosaics with locus-specific, stably-integrated transgenic elements | |
| WO2025223110A1 (en) | A method of generating a functional mch neurons | |
| Grandela et al. | STRAIGHT-IN: A platform for high-throughput targeting of large DNA payloads into human pluripotent stem cells | |
| Thorsen | The origin and properties of pro-oncogenic fields in the intestinal epithelium | |
| WO2021242989A1 (en) | Compositions and methods for inducing cas9 protein expression by use of autonomous pd-1 promoter activity | |
| Karayol et al. | Serotonin receptor 4 in mature excitatory hippocampal neurons modulates mood and anxiety | |
| Distasio | Novel Regulators of Neural Crest and Neural Progenitor Survival | |
| Platt | Applications of genome editing for disease modeling in mice | |
| EP2455466A1 (en) | Dna fragment and pharmaceutical composition |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20180829 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20191219 |
|
| A621 | Written request for application examination |
Free format text: JAPANESE INTERMEDIATE CODE: A621 Effective date: 20191219 |
|
| A977 | Report on retrieval |
Free format text: JAPANESE INTERMEDIATE CODE: A971007 Effective date: 20201125 |
|
| A131 | Notification of reasons for refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A131 Effective date: 20201203 |
|
| A601 | Written request for extension of time |
Free format text: JAPANESE INTERMEDIATE CODE: A601 Effective date: 20210225 |
|
| RD04 | Notification of resignation of power of attorney |
Free format text: JAPANESE INTERMEDIATE CODE: A7424 Effective date: 20210308 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20210601 |
|
| A02 | Decision of refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A02 Effective date: 20211111 |