JP2024525665A - 効率的なゲノム編集のための組成物及び方法 - Google Patents
効率的なゲノム編集のための組成物及び方法 Download PDFInfo
- Publication number
- JP2024525665A JP2024525665A JP2024501179A JP2024501179A JP2024525665A JP 2024525665 A JP2024525665 A JP 2024525665A JP 2024501179 A JP2024501179 A JP 2024501179A JP 2024501179 A JP2024501179 A JP 2024501179A JP 2024525665 A JP2024525665 A JP 2024525665A
- Authority
- JP
- Japan
- Prior art keywords
- seq
- sequence
- domain
- prime editing
- editing composition
- 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
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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/10—Transferases (2.)
- C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
- C12N9/1241—Nucleotidyltransferases (2.7.7)
- C12N9/1276—RNA-directed DNA polymerase (2.7.7.49), i.e. reverse transcriptase or telomerase
-
- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/62—DNA sequences coding for fusion proteins
-
- 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]
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
-
- 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
- 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
- C12N15/113—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
-
- 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
-
- 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]
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Crystallography & Structural Chemistry (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Peptides Or Proteins (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)
- Apparatus Associated With Microorganisms And Enzymes (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163218744P | 2021-07-06 | 2021-07-06 | |
| US63/218,744 | 2021-07-06 | ||
| US202163219623P | 2021-07-08 | 2021-07-08 | |
| US63/219,623 | 2021-07-08 | ||
| PCT/US2022/035613 WO2023283092A1 (en) | 2021-07-06 | 2022-06-29 | Compositions and methods for efficient genome editing |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2024525665A true JP2024525665A (ja) | 2024-07-12 |
| JPWO2023283092A5 JPWO2023283092A5 (https=) | 2025-07-07 |
| JP2024525665A5 JP2024525665A5 (https=) | 2025-07-07 |
Family
ID=84800962
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024501179A Pending JP2024525665A (ja) | 2021-07-06 | 2022-06-29 | 効率的なゲノム編集のための組成物及び方法 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20240228988A1 (https=) |
| EP (1) | EP4367227A4 (https=) |
| JP (1) | JP2024525665A (https=) |
| AU (1) | AU2022306377A1 (https=) |
| CA (1) | CA3224970A1 (https=) |
| WO (1) | WO2023283092A1 (https=) |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP7657726B2 (ja) | 2019-03-19 | 2025-04-07 | ザ ブロード インスティテュート，インコーポレーテッド | 編集ヌクレオチド配列を編集するための方法および組成物 |
| CA3174483A1 (en) | 2020-03-04 | 2021-09-10 | Flagship Pioneering Innovations Vi, Llc | Improved methods and compositions for modulating a genome |
| DE112021002672T5 (de) | 2020-05-08 | 2023-04-13 | President And Fellows Of Harvard College | Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz |
| JP2024533311A (ja) | 2021-09-08 | 2024-09-12 | フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー | ゲノムを調節するための方法及び組成物 |
| KR20240099164A (ko) | 2021-09-08 | 2024-06-28 | 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 | Pah-조절 조성물 및 방법 |
| JP2024534945A (ja) * | 2021-09-10 | 2024-09-26 | アジレント・テクノロジーズ・インク | 化学修飾を有するプライム編集のためのガイドｒｎａ |
| EP4444362A4 (en) | 2021-12-10 | 2026-04-01 | Flagship Pioneering Innovations Vi Llc | CFTR COMPOSITIONS AND MODULATION METHODS |
| WO2023225670A2 (en) | 2022-05-20 | 2023-11-23 | Tome Biosciences, Inc. | Ex vivo programmable gene insertion |
| WO2024020587A2 (en) | 2022-07-22 | 2024-01-25 | Tome Biosciences, Inc. | Pleiopluripotent stem cell programmable gene insertion |
| EP4665865A1 (en) | 2023-02-17 | 2025-12-24 | Anjarium Biosciences AG | Methods of making dna molecules and compositions and uses thereof |
| WO2024178144A1 (en) * | 2023-02-22 | 2024-08-29 | Prime Medicine, Inc. | Methods and compositions for editing nucleotide sequences |
| EP4720304A2 (en) * | 2023-05-31 | 2026-04-08 | University of Massachusetts | Improved modular prime editing with modified effectors and templates |
| WO2024259051A1 (en) * | 2023-06-14 | 2024-12-19 | The Children's Medical Center Corporation | Systems and methods for modifying a polynucleotide |
| WO2025038881A1 (en) * | 2023-08-16 | 2025-02-20 | Beam Therapeutics Inc. | Prime editing of single base mutations in sickle cell disease |
| WO2025076306A1 (en) * | 2023-10-06 | 2025-04-10 | University Of Massachusetts | Prime editors having improved prime editing efficiency |
| US20250354138A1 (en) | 2024-03-15 | 2025-11-20 | Beam Therapeutics Inc. | Prime editing of single base mutations in alpha-1 antitrypsin deficiency |
| WO2025226946A1 (en) * | 2024-04-24 | 2025-10-30 | Cedric Francois | Methods and compositions for the treatment of androgenic alopecia |
| WO2025231071A1 (en) * | 2024-05-01 | 2025-11-06 | Beam Therapeutics Inc. | Compositions and methods for cell conditioning |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2019186348A1 (en) * | 2018-03-25 | 2019-10-03 | GeneTether, Inc | Modified nucleic acid editing systems for tethering donor dna |
| WO2020033083A1 (en) * | 2018-08-10 | 2020-02-13 | Cornell University | Optimized base editors enable efficient editing in cells, organoids and mice |
| KR20210121113A (ko) * | 2019-01-31 | 2021-10-07 | 빔 테라퓨틱스, 인크. | 비-표적 탈아미노화가 감소된 핵염기 편집기 및 핵염기 편집기를 특성규명하기 위한 분석 |
| CN116355966A (zh) * | 2019-02-02 | 2023-06-30 | 上海科技大学 | 一种融合蛋白在遗传编辑的用途 |
| JP7657726B2 (ja) * | 2019-03-19 | 2025-04-07 | ザ ブロード インスティテュート，インコーポレーテッド | 編集ヌクレオチド配列を編集するための方法および組成物 |
| WO2021042047A1 (en) * | 2019-08-30 | 2021-03-04 | The General Hospital Corporation | C-to-g transversion dna base editors |
| EP4081635A4 (en) * | 2019-12-26 | 2024-03-27 | Agency for Science, Technology and Research | Nucleobase editors |
-
2022
- 2022-06-29 WO PCT/US2022/035613 patent/WO2023283092A1/en not_active Ceased
- 2022-06-29 CA CA3224970A patent/CA3224970A1/en active Pending
- 2022-06-29 JP JP2024501179A patent/JP2024525665A/ja active Pending
- 2022-06-29 AU AU2022306377A patent/AU2022306377A1/en active Pending
- 2022-06-29 EP EP22838255.2A patent/EP4367227A4/en active Pending
-
2024
- 2024-01-04 US US18/404,456 patent/US20240228988A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| US20240228988A1 (en) | 2024-07-11 |
| EP4367227A4 (en) | 2025-04-30 |
| WO2023283092A1 (en) | 2023-01-12 |
| CA3224970A1 (en) | 2023-01-12 |
| AU2022306377A1 (en) | 2024-01-25 |
| EP4367227A1 (en) | 2024-05-15 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US20240228988A1 (en) | Compositions and methods for efficient genome editing | |
| US20240011007A1 (en) | Genome editing compositions and methods for treatment of chronic granulomatous disease | |
| US20240067940A1 (en) | Methods and compositions for editing nucleotide sequences | |
| US20240424138A1 (en) | Genome editing compositions and method for treatment of retinitis pigmentosa | |
| US20250297246A1 (en) | Modified prime editing guide rnas | |
| AU2022283964A1 (en) | Genome editing compositions and methods for treatment of wilson's disease | |
| US20240229038A1 (en) | Genome editing compositions and methods for treatment of wilson's disease | |
| US20240360476A1 (en) | Genome Editing Compositions and Methods for Treatment of Myotonic Dystrophy | |
| EP4504921A2 (en) | Methods and compositions for editing nucleotide sequences | |
| EP4419688A2 (en) | Genome editing compositions and methods for treatment of usher syndrome type 3 | |
| US20250011766A1 (en) | Genome Editing Compositions and Methods for Treatment of Amyotrophic Lateral Sclerosis | |
| EP4430185A1 (en) | Genome editing compositions and methods for treatment of fuchs endothelial corneal dystrophy | |
| WO2024178144A1 (en) | Methods and compositions for editing nucleotide sequences | |
| US20240376466A1 (en) | Genome editing compositions and methods for treatment of fanconi anemia | |
| EP4658781A2 (en) | Genome editing compositions and methods for treatment of cystic fibrosis | |
| US20250354138A1 (en) | Prime editing of single base mutations in alpha-1 antitrypsin deficiency | |
| WO2025038881A1 (en) | Prime editing of single base mutations in sickle cell disease | |
| CN117999347A (zh) | 用于高效基因组编辑的组合物和方法 | |
| AU2024215960A1 (en) | Genome editing compositions and methods for treatment of cystic fibrosis | |
| WO2025090637A2 (en) | Genome editing compositions and methods for treatment of retinitis pigmentosa | |
| EP4437102A1 (en) | Genome editing compositions and methods for treatment of glycogen storage disease type 1b |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20250627 |
|
| A621 | Written request for application examination |
Free format text: JAPANESE INTERMEDIATE CODE: A621 Effective date: 20250627 |