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 PDF

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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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ncas9
cas9
terminal fragment
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amino acid
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CN112969790A (zh
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D·布赖森
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Bim Medical Co ltd
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    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62DNA sequences coding for fusion proteins
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    • C12N15/113Non-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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    • C12N15/90Stable introduction of foreign DNA into chromosome
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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N9/14Hydrolases (3)
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    • C12Y305/00Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04002Adenine deaminase (3.5.4.2)
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    • C12Y305/04004Adenosine deaminase (3.5.4.4)
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    • C07K2319/00Fusion polypeptide
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    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
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    • C07K2319/00Fusion polypeptide
    • C07K2319/90Fusion polypeptide containing a motif for post-translational modification
    • C07K2319/92Fusion polypeptide containing a motif for post-translational modification containing an intein ("protein splicing")domain
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
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    • C12N2800/00Nucleic acids vectors
    • C12N2800/80Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Wood Science & Technology (AREA)
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  • 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)
CN201980073055.6A 2018-09-07 2019-09-07 用于递送核碱基编辑系统的组合物和方法 Active CN112969790B (zh)

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

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CN112969790B true CN112969790B (zh) 2024-07-05

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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)
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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
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GB2632565B (en) * 2020-04-09 2025-06-04 Verve Therapeutics Inc Base editing of PCSK9 and methods of using same for treatment of disease
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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)
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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
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WO2024233941A1 (en) * 2023-05-11 2024-11-14 Massachusetts Eye And Ear Infirmary Base editing approaches to treat abca4-associated stargardt disease
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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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