CN114072509B - 脱氨反应脱靶减低的核碱基编辑器和使用其修饰核碱基靶序列的方法 - Google Patents

脱氨反应脱靶减低的核碱基编辑器和使用其修饰核碱基靶序列的方法 Download PDF

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CN114072509B
CN114072509B CN202080026310.4A CN202080026310A CN114072509B CN 114072509 B CN114072509 B CN 114072509B CN 202080026310 A CN202080026310 A CN 202080026310A CN 114072509 B CN114072509 B CN 114072509B
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fusion protein
cytidine
amino acid
base editor
cas9
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CN114072509A (zh
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N·戈代尔利
Y·于
I·斯雷梅克
J·M·格尔克
S-J·李
D·A·玻恩
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Bim Medical Co ltd
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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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    • 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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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2497—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing N- glycosyl compounds (3.2.2)
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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)
    • C12N9/80—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5) acting on amide bonds in linear amides (3.5.1)
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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)
    • C12Y305/04005—Cytidine deaminase (3.5.4.5)
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    • C07K2319/00—Fusion polypeptide
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    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
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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
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    • 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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CN202080026310.4A 2019-01-31 2020-01-31 脱氨反应脱靶减低的核碱基编辑器和使用其修饰核碱基靶序列的方法 Active CN114072509B (zh)

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US201962799702P 2019-01-31 2019-01-31
US62/799,702 2019-01-31
US201962835456P 2019-04-17 2019-04-17
US62/835,456 2019-04-17
US201962941569P 2019-11-27 2019-11-27
US62/941,569 2019-11-27
PCT/US2020/016288 WO2020160517A1 (en) 2019-01-31 2020-01-31 Nucleobase editors having reduced off-target deamination and methods of using same to modify a nucleobase target sequence

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CN202510365522.XA Pending CN120400115A (zh) 2019-01-31 2020-01-31 脱氨反应脱靶减低的核碱基编辑器和使用其修饰核碱基靶序列的方法

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JP (2) JP7600122B2 (https=)
KR (1) KR20210124280A (https=)
CN (2) CN114072509B (https=)
AU (1) AU2020216484A1 (https=)
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EP3847254A4 (en) 2018-09-07 2022-08-10 Beam Therapeutics Inc. COMPOSITIONS AND METHODS FOR DELIVERING A NUCLEOBASE EDITING SYSTEM
WO2020051562A2 (en) 2018-09-07 2020-03-12 Beam Therapeutics Inc. Compositions and methods for improving base editing
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
AU2020215730A1 (en) 2019-01-31 2021-07-29 Beam Therapeutics Inc. Nucleobase editors having reduced non-target deamination and assays for characterizing nucleobase editors
CN120174005A (zh) 2019-02-13 2025-06-20 比姆医疗股份有限公司 具有用于修饰靶标序列中核碱基的腺苷脱氨酶碱基编辑器的经修饰的免疫细胞
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CN120400115A (zh) 2025-08-01
CA3127494A1 (en) 2020-08-06
JP2022521460A (ja) 2022-04-08
KR20210124280A (ko) 2021-10-14
WO2020160517A1 (en) 2020-08-06
EP3918077A1 (en) 2021-12-08
EP3918077A4 (en) 2023-03-29
AU2020216484A1 (en) 2021-07-29
CN114072509A (zh) 2022-02-18
JP2025041661A (ja) 2025-03-26
JP7779988B2 (ja) 2025-12-03
JP7600122B2 (ja) 2024-12-16
US20220136012A1 (en) 2022-05-05

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