JP7541508B2 - 操作された標的特異的な塩基エディター - Google Patents

操作された標的特異的な塩基エディター Download PDF

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JP7541508B2
JP7541508B2 JP2021510000A JP2021510000A JP7541508B2 JP 7541508 B2 JP7541508 B2 JP 7541508B2 JP 2021510000 A JP2021510000 A JP 2021510000A JP 2021510000 A JP2021510000 A JP 2021510000A JP 7541508 B2 JP7541508 B2 JP 7541508B2
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dna
domain
editing
base
nickase
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JP2021536229A (ja
JP2021536229A5 (enExample
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フリードリック・ファウザー
ジェフリー・シー・ミラー
エドワード・リバー
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Sangamo Therapeutics Inc
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
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    • 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)
    • C12Y305/04001—Cytosine deaminase (3.5.4.1)
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    • 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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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3181—Peptide nucleic acid, PNA
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
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    • C12Q2521/00—Reaction characterised by the enzymatic activity
    • C12Q2521/30—Phosphoric diester hydrolysing, i.e. nuclease
    • C12Q2521/307—Single strand endonuclease
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    • C12Q2521/00—Reaction characterised by the enzymatic activity
    • C12Q2521/50—Other enzymatic activities
    • C12Q2521/539—Deaminase
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    • C12Q2522/00—Reaction characterised by the use of non-enzymatic proteins
    • C12Q2522/10—Nucleic acid binding proteins
    • C12Q2522/101—Single or double stranded nucleic acid binding proteins

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2021510000A 2018-08-23 2019-08-20 操作された標的特異的な塩基エディター Active JP7541508B2 (ja)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201862721903P 2018-08-23 2018-08-23
US62/721,903 2018-08-23
US201862753696P 2018-10-31 2018-10-31
US62/753,696 2018-10-31
US201962817153P 2019-03-12 2019-03-12
US62/817,153 2019-03-12
US201962867565P 2019-06-27 2019-06-27
US62/867,565 2019-06-27
PCT/US2019/047172 WO2020041249A1 (en) 2018-08-23 2019-08-20 Engineered target specific base editors

Publications (3)

Publication Number Publication Date
JP2021536229A JP2021536229A (ja) 2021-12-27
JP2021536229A5 JP2021536229A5 (enExample) 2022-08-29
JP7541508B2 true JP7541508B2 (ja) 2024-08-28

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JP2021510000A Active JP7541508B2 (ja) 2018-08-23 2019-08-20 操作された標的特異的な塩基エディター

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US (1) US11834686B2 (enExample)
EP (1) EP3841204A4 (enExample)
JP (1) JP7541508B2 (enExample)
KR (1) KR102712986B1 (enExample)
CN (1) CN112867792A (enExample)
AU (1) AU2019326408A1 (enExample)
CA (1) CA3109592A1 (enExample)
IL (1) IL280951B2 (enExample)
SG (1) SG11202101801RA (enExample)
WO (1) WO2020041249A1 (enExample)

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AU2019326408A1 (en) 2018-08-23 2021-03-11 Sangamo Therapeutics, Inc. Engineered target specific base editors
JP7059965B2 (ja) * 2019-02-21 2022-04-26 株式会社豊田中央研究所 エピゲノム状態を制御する融合タンパク質及びその利用
WO2021155166A1 (en) * 2020-01-31 2021-08-05 University Of Massachusetts A cas9-pdbd base editor platform with improved targeting range and specificity
CN111518794B (zh) * 2020-04-13 2023-05-16 中山大学 基于激活诱导性胞苷脱氨酶的诱导突变蛋白的制备和用途
CN111690720B (zh) * 2020-06-16 2021-06-15 山东舜丰生物科技有限公司 利用修饰的单链核酸进行靶核酸检测的方法
CA3187135A1 (en) * 2020-06-30 2022-01-06 Pairwise Plants Services, Inc. Compositions, systems, and methods for base diversification
US20230340486A1 (en) * 2020-07-27 2023-10-26 The Children’S Hospital Of Philadelphia In utero and postnatal gene editing and therapy for treatment of monogenic diseases, including mucopolysaccharidosis type 1h and other disorders
CN116261594A (zh) * 2020-09-25 2023-06-13 桑格摩生物治疗股份有限公司 用于核碱基编辑的锌指融合蛋白
CA3196347A1 (en) * 2020-10-23 2022-04-28 Jonathan Thon Compositions and methods related to megakaryocyte-derived extracellular vesicles for treating myeloproliferative neoplasms
CN117015552A (zh) * 2020-10-23 2023-11-07 斯特姆生物公司 用于治疗骨髓增殖性肿瘤的与巨核细胞来源的细胞外囊泡相关的组合物和方法
IL303506A (en) * 2020-12-11 2023-08-01 Intellia Therapeutics Inc Polynucleotides, compositions, and methods for genome editing involving deamination
CA3218511A1 (en) 2021-05-10 2022-11-17 Sqz Biotechnologies Company Methods for delivering genome editing molecules to the nucleus or cytosol of a cell and uses thereof
WO2022271548A2 (en) * 2021-06-23 2022-12-29 Massachusetts Institute Of Technology Compositions, methods and systems for the delivery of gene editing material to cells
AU2022317661B2 (en) * 2021-07-26 2026-04-23 Dana-Farber Cancer Institute, Inc. Small molecule inhibition of deubiquitinating enzyme josephin domain containing 1 (josd1) as a targeted therapy for leukemias with mutant janus kinase 2 (jak2)
US20240417755A1 (en) * 2021-09-27 2024-12-19 Vor Biopharma Inc. Fusion polypeptides for genetic editing and methods of use thereof
US20240415979A1 (en) * 2021-10-23 2024-12-19 Strm.Bio Incorporated Gene editing to treat myeloproliferative neoplasms
JP2025504606A (ja) * 2021-12-22 2025-02-13 サンガモ セラピューティクス， インコーポレイテッド 核酸塩基編集のための新規ジンクフィンガー融合タンパク質
CN116497002A (zh) * 2022-01-19 2023-07-28 中国科学院动物研究所 工程化的CasX核酸酶、效应蛋白及其用途
EP4482855A4 (en) * 2022-02-22 2026-03-04 Peter Biotherapeutics Inc GENE MODIFICATION TECHNOLOGIES
JP2023131616A (ja) * 2022-03-09 2023-09-22 国立大学法人広島大学 Ｄｎａ編集システム、並びに、それを用いた標的ｄｎａの編集方法及び標的ｄｎａが編集された細胞の製造方法
KR20230139064A (ko) * 2022-03-25 2023-10-05 기초과학연구원 식물세포 소기관 dna의 아데닌을 구아닌으로 염기 교정
CN119923471A (zh) * 2022-09-21 2025-05-02 基础科学研究院 植物细胞器dna的碱基编辑
JP2025534400A (ja) * 2022-09-30 2025-10-15 北京昌平▲実▼▲験▼室 二本鎖ｄｎａデアミナーゼを用いたフットプリンティングによりゲノムワイドｄｎａ結合タンパク質結合部位を決定するための方法
CN117384885B (zh) * 2022-12-15 2025-06-10 中国科学院遗传与发育生物学研究所 一种碱基编辑器及其应用
JP2026501648A (ja) * 2022-12-30 2026-01-16 北京大学 塩基エディターシステムおよびその使用方法
WO2024155727A1 (en) * 2023-01-17 2024-07-25 The Broad Institute, Inc. Systems and methods for targeted continuous genome mutagenesis
WO2025259055A1 (ko) * 2024-06-13 2025-12-18 주식회사 엣진 징크 핑거 단백질 및 닉카제를 포함하는 신규한 염기 에디터

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