CN112969790B - 用于递送核碱基编辑系统的组合物和方法 - Google Patents
用于递送核碱基编辑系统的组合物和方法 Download PDFInfo
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- CN112969790B CN112969790B CN201980073055.6A CN201980073055A CN112969790B CN 112969790 B CN112969790 B CN 112969790B CN 201980073055 A CN201980073055 A CN 201980073055A CN 112969790 B CN112969790 B CN 112969790B
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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- C—CHEMISTRY; METALLURGY
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- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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- C—CHEMISTRY; METALLURGY
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- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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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- 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]
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
- C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
- C12Y305/04002—Adenine deaminase (3.5.4.2)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
- C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
- C12Y305/04004—Adenosine deaminase (3.5.4.4)
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
-
- 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
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/90—Fusion polypeptide containing a motif for post-translational modification
- C07K2319/92—Fusion polypeptide containing a motif for post-translational modification containing an intein ("protein splicing")domain
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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]
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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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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- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Medicinal Chemistry (AREA)
- Mycology (AREA)
- Cell Biology (AREA)
- Crystallography & Structural Chemistry (AREA)
- Enzymes And Modification Thereof (AREA)
- Peptides Or Proteins (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862728703P | 2018-09-07 | 2018-09-07 | |
| US62/728,703 | 2018-09-07 | ||
| US201862779404P | 2018-12-13 | 2018-12-13 | |
| US62/779,404 | 2018-12-13 | ||
| PCT/US2019/050111 WO2020051561A1 (en) | 2018-09-07 | 2019-09-07 | Compositions and methods for delivering a nucleobase editing system |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| CN112969790A CN112969790A (zh) | 2021-06-15 |
| CN112969790B true CN112969790B (zh) | 2024-07-05 |
Family
ID=69722796
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201980073055.6A Active CN112969790B (zh) | 2018-09-07 | 2019-09-07 | 用于递送核碱基编辑系统的组合物和方法 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20210301274A1 (enExample) |
| EP (1) | EP3847254A4 (enExample) |
| JP (2) | JP7657711B2 (enExample) |
| KR (1) | KR20210055733A (enExample) |
| CN (1) | CN112969790B (enExample) |
| AU (1) | AU2019336245A1 (enExample) |
| CA (1) | CA3112011A1 (enExample) |
| WO (1) | WO2020051561A1 (enExample) |
Families Citing this family (28)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11649442B2 (en) | 2017-09-08 | 2023-05-16 | The Regents Of The University Of California | RNA-guided endonuclease fusion polypeptides and methods of use thereof |
| JP7642531B2 (ja) | 2018-05-11 | 2025-03-10 | ビーム セラピューティクス インク. | プログラム可能塩基エディターシステムを用いて病原性アミノ酸を置換する方法 |
| KR20210055733A (ko) * | 2018-09-07 | 2021-05-17 | 빔 테라퓨틱스, 인크. | 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법 |
| WO2020051562A2 (en) | 2018-09-07 | 2020-03-12 | Beam Therapeutics Inc. | Compositions and methods for improving base editing |
| JP2022536364A (ja) | 2019-06-13 | 2022-08-15 | ザ ジェネラル ホスピタル コーポレイション | 操作されたヒト内在性ウイルス様粒子および細胞への送達のためのその使用方法 |
| WO2021016075A1 (en) | 2019-07-19 | 2021-01-28 | Flagship Pioneering Innovations Vi, Llc | Recombinase compositions and methods of use |
| MX2022010991A (es) | 2020-03-04 | 2023-02-09 | Flagship Pioneering Innovations Vi Llc | Metodos y composiciones para modular un genoma. |
| MX2022011562A (es) | 2020-03-19 | 2022-12-13 | Intellia Therapeutics Inc | Métodos y composiciones para edición dirigida de genoma. |
| GB2632565B (en) * | 2020-04-09 | 2025-06-04 | Verve Therapeutics Inc | Base editing of PCSK9 and methods of using same for treatment of disease |
| WO2022020800A2 (en) | 2020-07-24 | 2022-01-27 | The General Hospital Corporation | Enhanced virus-like particles and methods of use thereof for delivery to cells |
| US20230383277A1 (en) * | 2020-10-14 | 2023-11-30 | Beam Therapeutics Inc. | Compositions and methods for treating glycogen storage disease type 1a |
| WO2023010135A1 (en) | 2021-07-30 | 2023-02-02 | Tune Therapeutics, Inc. | Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) |
| EP4377459A2 (en) | 2021-07-30 | 2024-06-05 | Tune Therapeutics, Inc. | Compositions and methods for modulating expression of frataxin (fxn) |
| 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 |
| JP2024544013A (ja) | 2021-12-03 | 2024-11-26 | ザ ブロード インスティテュート,インコーポレーテッド | 有効なin vivo送達のための組成物および方法 |
| CN117187220A (zh) * | 2022-03-08 | 2023-12-08 | 中国科学院遗传与发育生物学研究所 | 腺嘌呤脱氨酶及其在碱基编辑中的用途 |
| CN120303407A (zh) | 2022-05-17 | 2025-07-11 | 恩维洛普治疗有限责任公司 | 用于有效体内递送的组合物和方法 |
| EP4554967A2 (en) | 2022-07-12 | 2025-05-21 | Tune Therapeutics, Inc. | Compositions, systems, and methods for targeted transcriptional activation |
| WO2024097763A1 (en) * | 2022-11-01 | 2024-05-10 | Memorial Sloan-Kettering Cancer Center | Intein-based sorting system and modular chimeric polypeptides |
| WO2024163683A2 (en) | 2023-02-01 | 2024-08-08 | Tune Therapeutics, Inc. | Systems, compositions, and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) and x-inactive specific transcript (xist) |
| WO2024163678A2 (en) | 2023-02-01 | 2024-08-08 | Tune Therapeutics, Inc. | Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods |
| WO2024233941A1 (en) * | 2023-05-11 | 2024-11-14 | Massachusetts Eye And Ear Infirmary | Base editing approaches to treat abca4-associated stargardt disease |
| WO2024249954A1 (en) | 2023-05-31 | 2024-12-05 | Capstan Therapeutics, Inc. | Lipid nanoparticle formulations and compositions |
| WO2025038955A1 (en) * | 2023-08-16 | 2025-02-20 | Seattle Children's Hospital D/B/A Seattle Children's Research Institute | Systems and methods for improving safety of split intein aav mediated gene therapy |
| CN117568313B (zh) * | 2024-01-15 | 2024-04-26 | 上海贝斯昂科生物科技有限公司 | 基因编辑组合物及其用途 |
| WO2025194138A1 (en) | 2024-03-14 | 2025-09-18 | Tessera Therapeutics, Inc. | St1cas9 compositions and methods for modulating a genome |
| WO2025194124A1 (en) | 2024-03-14 | 2025-09-18 | Tessera Therapeutics, Inc. | Modified st1cas9 guide nucleic acids |
| WO2025243196A1 (en) * | 2024-05-21 | 2025-11-27 | Fondazione Istituto Italiano Di Tecnologia | Gene regulation system (tuser) |
Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2016205759A1 (en) * | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG11201609211VA (en) * | 2014-03-05 | 2016-12-29 | Nat Univ Corp Univ Kobe | Genomic sequence modification method for specifically converting nucleic acid bases of targeted dna sequence, and molecular complex for use in same |
| WO2016022363A2 (en) * | 2014-07-30 | 2016-02-11 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| US20180155708A1 (en) | 2015-01-08 | 2018-06-07 | President And Fellows Of Harvard College | Split Cas9 Proteins |
| CN106011104B (zh) | 2015-05-21 | 2019-09-27 | 清华大学 | 利用拆分Cas系统进行基因编辑和表达调控方法 |
| WO2018035503A1 (en) | 2016-08-18 | 2018-02-22 | The Regents Of The University Of California | Crispr-cas genome engineering via a modular aav delivery system |
| JP7588390B2 (ja) * | 2016-10-14 | 2024-11-22 | 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 | 核酸塩基エディターのaav送達 |
| KR20210055733A (ko) * | 2018-09-07 | 2021-05-17 | 빔 테라퓨틱스, 인크. | 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법 |
-
2019
- 2019-09-07 KR KR1020217009863A patent/KR20210055733A/ko active Pending
- 2019-09-07 CA CA3112011A patent/CA3112011A1/en active Pending
- 2019-09-07 US US17/273,209 patent/US20210301274A1/en active Pending
- 2019-09-07 JP JP2021512514A patent/JP7657711B2/ja active Active
- 2019-09-07 WO PCT/US2019/050111 patent/WO2020051561A1/en not_active Ceased
- 2019-09-07 AU AU2019336245A patent/AU2019336245A1/en active Pending
- 2019-09-07 EP EP19857653.0A patent/EP3847254A4/en active Pending
- 2019-09-07 CN CN201980073055.6A patent/CN112969790B/zh active Active
-
2024
- 2024-11-01 JP JP2024192680A patent/JP2025028857A/ja active Pending
Patent Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2016205759A1 (en) * | 2015-06-18 | 2016-12-22 | The Broad Institute Inc. | Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation |
Also Published As
| Publication number | Publication date |
|---|---|
| JP2025028857A (ja) | 2025-03-05 |
| KR20210055733A (ko) | 2021-05-17 |
| JP7657711B2 (ja) | 2025-04-07 |
| EP3847254A4 (en) | 2022-08-10 |
| AU2019336245A1 (en) | 2021-04-01 |
| CA3112011A1 (en) | 2020-03-12 |
| WO2020051561A1 (en) | 2020-03-12 |
| CN112969790A (zh) | 2021-06-15 |
| JP2022500017A (ja) | 2022-01-04 |
| EP3847254A1 (en) | 2021-07-14 |
| US20210301274A1 (en) | 2021-09-30 |
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