AR114014A1 - Sistema de edición de bases y método basado en proteína cpf1 - Google Patents

Sistema de edición de bases y método basado en proteína cpf1

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Publication number
AR114014A1
AR114014A1 ARP180103817A ARP180103817A AR114014A1 AR 114014 A1 AR114014 A1 AR 114014A1 AR P180103817 A ARP180103817 A AR P180103817A AR P180103817 A ARP180103817 A AR P180103817A AR 114014 A1 AR114014 A1 AR 114014A1
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AR
Argentina
Prior art keywords
base editing
fusion protein
guide rna
nucleotide sequence
sequence encoding
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ARP180103817A
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English (en)
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Inst Genetics & Developmental Biology Cas
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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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    • 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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    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/82—Vectors or expression systems specially adapted for eukaryotic hosts for plant cells, e.g. plant artificial chromosomes (PACs)
    • C12N15/8201—Methods for introducing genetic material into plant cells, e.g. DNA, RNA, stable or transient incorporation, tissue culture methods adapted for transformation
    • C12N15/8213—Targeted insertion of genes into the plant genome by homologous recombination
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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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/82—Vectors or expression systems specially adapted for eukaryotic hosts for plant cells, e.g. plant artificial chromosomes (PACs)
    • C12N15/8216—Methods for controlling, regulating or enhancing expression of transgenes in plant cells
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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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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    • 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
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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 RNAses, DNAses
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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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    • 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)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
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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 [CRISPRs]

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  • Health & Medical Sciences (AREA)
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  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
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  • Biophysics (AREA)
  • Cell Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Mycology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Breeding Of Plants And Reproduction By Means Of Culturing (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

La presente se relaciona con el campo de la ingeniería genética. En particular, la presente se relaciona con un sistema y método para la edición de bases basado en la proteína CPF1. Más particularmente, la presente se relaciona con un sistema y método para la edición eficaz de bases de una secuencia objetivo en el genoma de un organismo (por ejemplo, una planta) a través de una proteína de fusión CpfI-desaminasa dirigida por ARN guía, y el organismo genéticamente modificado (por ejemplo, plantas) producido por el método y su progenie. Reivindicación 1: Un sistema para la edición de bases de una secuencia objetivo en el genoma de un organismo, que comprende por lo menos uno de los siguientes puntos i) a v): i) una proteína de fusión para la edición de bases, y un ARN guía; ii) una construcción de expresión que comprende una secuencia de nucleótidos que codifica una proteína de fusión para la edición de bases, y un ARN guía; iii) una proteína de fusión para la edición de bases, y una construcción de expresión que comprende una secuencia de nucleótidos que codifican un ARN guía; iv) una construcción de expresión que comprende una secuencia de nucleótidos que codifica una proteína de fusión para la edición de bases, y una construcción de expresión que comprende una secuencia de nucleótidos que codifica un ARN guía; v) una construcción de expresión que comprende una secuencia de nucleótidos que codifica una proteína de fusión para la edición de bases y una secuencia de nucleótidos que codifica un ARN guía; en donde la proteína de fusión para la edición de bases comprende una Cpf1 que carece de actividad de escisión de ADN y una desamina, el ARN guía es capaz de dirigir la proteína de fusión para la edición de bases a una secuencia objetivo en el genoma, que resulta en una o más sustituciones de C a T o de A a G en la secuencia objetivo.
ARP180103817A 2017-12-22 2018-12-21 Sistema de edición de bases y método basado en proteína cpf1 AR114014A1 (es)

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CN201711403490 2017-12-22

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AR (1) AR114014A1 (es)
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WO2020168133A1 (en) * 2019-02-13 2020-08-20 Beam Therapeutics Inc. Compositions and methods for treating hemoglobinopathies
WO2021016086A1 (en) * 2019-07-19 2021-01-28 Pairwise Plants Services, Inc. Optimized protein linkers and methods of use
CN114945670A (zh) * 2019-08-20 2022-08-26 上海蓝十字医学科学研究所 一种碱基编辑***和其使用方法
CN110551752B (zh) * 2019-08-30 2023-03-14 北京市农林科学院 xCas9n-epBE碱基编辑***及其在基因组碱基替换中的应用
WO2021050571A1 (en) * 2019-09-09 2021-03-18 Beam Therapeutics Inc. Novel nucleobase editors and methods of using same
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WO2021056302A1 (en) * 2019-09-26 2021-04-01 Syngenta Crop Protection Ag Methods and compositions for dna base editing
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CN111019967A (zh) * 2019-11-27 2020-04-17 南京农业大学 GmU3-19g-1和GmU6-16g-1启动子在大豆多基因编辑***中的应用
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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
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CN114317589B (zh) * 2020-09-30 2024-01-16 北京市农林科学院 SpRYn-ABE碱基编辑***在植物基因组碱基替换中的应用
CN114317596B (zh) * 2020-09-30 2024-01-16 北京市农林科学院 一种将植物基因组靶点序列中的a突变为g的方法
CN112430622A (zh) * 2020-10-26 2021-03-02 扬州大学 一种FokI和dCpf1融合蛋白表达载体及其介导的定点基因编辑方法
CN113005141A (zh) * 2021-01-05 2021-06-22 温州医科大学 高活性突变体构成的基因编辑工具及制备方法和修复先天性视网膜劈裂症致病基因的方法
CN115216486B (zh) * 2021-04-21 2024-04-16 浙江大学 一种负链rna病毒载体及无需转化的植物基因组编辑方法
CN115704015A (zh) * 2021-08-12 2023-02-17 清华大学 基于腺嘌呤和胞嘧啶双碱基编辑器的靶向诱变***
CN114045302A (zh) * 2021-11-12 2022-02-15 三亚中国农业科学院国家南繁研究院 一种单碱基编辑载体及其构建和应用
CN114835818B (zh) * 2022-03-17 2024-03-22 江南大学 一种基因编辑融合蛋白、其构建的腺嘌呤碱基编辑器及其应用
WO2023187027A1 (en) * 2022-03-30 2023-10-05 BASF Agricultural Solutions Seed US LLC Optimized base editors
WO2023207607A1 (zh) * 2022-04-29 2023-11-02 北京大学 用于修饰线粒体dna的脱氨酶突变体、组合物和方法
CN114686456B (zh) * 2022-05-10 2023-02-17 中山大学 基于双分子脱氨酶互补的碱基编辑***及其应用
CN116376948B (zh) * 2022-07-25 2023-12-15 广州医科大学 一种质粒载体及展示外源蛋白的ms2噬菌体类似颗粒的制备方法
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CN116286734B (zh) * 2022-11-29 2024-04-02 武汉大学 野生型LbCas12a蛋白的突变体及SNP检测用途
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