JP7550057B2 - ドナーポリヌクレオチドの挿入によるゲノム編集のための組成物および方法 - Google Patents
ドナーポリヌクレオチドの挿入によるゲノム編集のための組成物および方法 Download PDFInfo
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- JP7550057B2 JP7550057B2 JP2020572386A JP2020572386A JP7550057B2 JP 7550057 B2 JP7550057 B2 JP 7550057B2 JP 2020572386 A JP2020572386 A JP 2020572386A JP 2020572386 A JP2020572386 A JP 2020572386A JP 7550057 B2 JP7550057 B2 JP 7550057B2
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- donor polynucleotide
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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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- 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
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- A—HUMAN NECESSITIES
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- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
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- 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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- 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]
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- C—CHEMISTRY; METALLURGY
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
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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
- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
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- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Medicinal Chemistry (AREA)
- Cell Biology (AREA)
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- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Crystallography & Structural Chemistry (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Apparatus Associated With Microorganisms And Enzymes (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862691573P | 2018-06-28 | 2018-06-28 | |
| US62/691,573 | 2018-06-28 | ||
| PCT/IB2019/000833 WO2020003006A2 (en) | 2018-06-28 | 2019-06-28 | Compositions and methods for genomic editing by insertion of donor polynucleotides |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021529518A JP2021529518A (ja) | 2021-11-04 |
| JP2021529518A5 JP2021529518A5 (enExample) | 2022-06-20 |
| JP7550057B2 true JP7550057B2 (ja) | 2024-09-12 |
Family
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Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020572386A Active JP7550057B2 (ja) | 2018-06-28 | 2019-06-28 | ドナーポリヌクレオチドの挿入によるゲノム編集のための組成物および方法 |
Country Status (12)
| Country | Link |
|---|---|
| US (3) | US11827877B2 (enExample) |
| EP (1) | EP3814499A2 (enExample) |
| JP (1) | JP7550057B2 (enExample) |
| KR (1) | KR20210027389A (enExample) |
| CN (1) | CN112585268B (enExample) |
| AU (1) | AU2019293286A1 (enExample) |
| BR (1) | BR112020025824A2 (enExample) |
| CA (1) | CA3104028A1 (enExample) |
| IL (1) | IL279718A (enExample) |
| MX (1) | MX2020014248A (enExample) |
| SG (1) | SG11202012499RA (enExample) |
| WO (1) | WO2020003006A2 (enExample) |
Families Citing this family (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3204496A1 (en) | 2014-10-10 | 2017-08-16 | Editas Medicine, Inc. | Compositions and methods for promoting homology directed repair |
| US11866727B2 (en) | 2015-11-06 | 2024-01-09 | Crispr Therapeutics Ag | Materials and methods for treatment of glycogen storage disease type 1A |
| EP3652312A1 (en) | 2017-07-14 | 2020-05-20 | Editas Medicine, Inc. | Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites |
| EP3814499A2 (en) | 2018-06-28 | 2021-05-05 | CRISPR Therapeutics AG | Compositions and methods for genomic editing by insertion of donor polynucleotides |
| IL326835A (en) | 2018-10-16 | 2026-04-01 | Blueallele Llc | Methods for targeted insertion of dna genes |
| 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 |
| US12521451B2 (en) | 2019-11-08 | 2026-01-13 | Regeneron Pharmaceuticals, Inc. | CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy |
| WO2021174168A1 (en) * | 2020-02-28 | 2021-09-02 | Blueallele, Llc | Methods for treating gain-of-function disorders combining gene editing and gene therapy |
| US12655422B2 (en) * | 2020-08-19 | 2026-06-16 | Integrated Dna Technologies, Inc. | Methods and compositions for inhibition of CRISPR re-cleavage events |
| US20240368587A1 (en) * | 2021-04-01 | 2024-11-07 | Duke University | Compositions For and Methods of Editing the Genome |
| CN113403309B (zh) * | 2021-05-24 | 2022-08-16 | 珠海舒桐医疗科技有限公司 | 非同源双链寡聚核苷酸片段在基因敲除系统中的应用 |
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- 2019-06-28 KR KR1020217002378A patent/KR20210027389A/ko not_active Ceased
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| US11332760B2 (en) | 2022-05-17 |
| AU2019293286A1 (en) | 2021-01-07 |
| BR112020025824A2 (pt) | 2021-03-23 |
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