KR20210058806A - Rna 표적화 융합 단백질 조성물 및 사용 방법 - Google Patents

Rna 표적화 융합 단백질 조성물 및 사용 방법 Download PDF

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KR20210058806A
KR20210058806A KR1020217000538A KR20217000538A KR20210058806A KR 20210058806 A KR20210058806 A KR 20210058806A KR 1020217000538 A KR1020217000538 A KR 1020217000538A KR 20217000538 A KR20217000538 A KR 20217000538A KR 20210058806 A KR20210058806 A KR 20210058806A
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rna
lys
glu
ala
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데이비드 에이 넬레스
란잔 바트라
유진 예오
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로카나바이오 인크.
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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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    • 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
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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
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    • C12N15/67—General methods for enhancing the expression
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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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    • 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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    • 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
    • C07K2319/85—Fusion polypeptide containing an RNA binding domain
    • 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
    • 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
    • C12N15/1131—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 against viruses
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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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    • 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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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)
KR1020217000538A 2018-06-08 2019-06-07 Rna 표적화 융합 단백질 조성물 및 사용 방법 Abandoned KR20210058806A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862682271P 2018-06-08 2018-06-08
US62/682,271 2018-06-08
PCT/US2019/036021 WO2019236982A1 (en) 2018-06-08 2019-06-07 Rna-targeting fusion protein compositions and methods for use

Publications (1)

Publication Number Publication Date
KR20210058806A true KR20210058806A (ko) 2021-05-24

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KR1020217000538A Abandoned KR20210058806A (ko) 2018-06-08 2019-06-07 Rna 표적화 융합 단백질 조성물 및 사용 방법

Country Status (9)

Country Link
US (3) US20200071718A1 (https=)
EP (1) EP3802812A4 (https=)
JP (1) JP2021526858A (https=)
KR (1) KR20210058806A (https=)
CN (1) CN112930395A (https=)
AU (1) AU2019280990A1 (https=)
CA (1) CA3102779A1 (https=)
SG (1) SG11202012004SA (https=)
WO (1) WO2019236982A1 (https=)

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WO2011160052A2 (en) 2010-06-18 2011-12-22 The University Of North Carolina At Chapel Hill Methods and compositions for sequence specific rna endonucleases
US20190284553A1 (en) 2018-03-15 2019-09-19 KSQ Therapeutics, Inc. Gene-regulating compositions and methods for improved immunotherapy
AU2020208346B2 (en) * 2019-01-14 2026-01-29 University Of Rochester Targeted nuclear RNA cleavage and polyadenylation with CRISPR-cas
US20220267773A1 (en) * 2019-07-23 2022-08-25 University Of Rochester Targeted RNA cleavage with CRISPR-Cas
US11572565B2 (en) 2020-07-21 2023-02-07 Trustees Of Boston University Inducible control of gene expression
CN112126645B (zh) * 2020-09-11 2021-06-01 广州吉赛生物科技股份有限公司 一种环形rna敲低方法及其应用
CN112430597A (zh) * 2020-11-24 2021-03-02 深圳市瑞吉生物科技有限公司 一种使目的基因沉默的CasRx制剂及其应用
WO2022119974A1 (en) * 2020-12-01 2022-06-09 Locanabio, Inc. Rna-targeting compositions and methods for treating cag repeat diseases
JP2023551874A (ja) * 2020-12-01 2023-12-13 ロックアネイビオ， インコーポレイテッド 筋強直性ジストロフィー１型を処置するためのｒｎａ標的化組成物および方法
GB202105455D0 (en) 2021-04-16 2021-06-02 Ucl Business Ltd Composition
WO2022256414A1 (en) * 2021-06-02 2022-12-08 The Regents Of The University Of California Rna recognition complex and uses thereof
CN118541475A (zh) * 2021-12-27 2024-08-23 亘喜生物科技（上海）有限公司 用于细胞修饰的系统和方法
CN114835776B (zh) * 2022-04-08 2023-09-01 陕西师范大学 一种靶向Smad4/PELO相互作用的抵抗肿瘤转移小分子多肽及应用
CN116949011A (zh) * 2022-04-26 2023-10-27 中国科学院动物研究所 经分离的Cas13蛋白、基于它的基因编辑系统及其用途
WO2024092230A2 (en) * 2022-10-28 2024-05-02 The Regents Of The University Of California Recombinant enhanced antiviral restrictors
CN115820603B (zh) * 2022-11-15 2024-07-05 吉林大学 一种基于dCasRx-NSUN6单基因特异性M5C修饰编辑方法

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Publication number Publication date
US10822617B2 (en) 2020-11-03
AU2019280990A1 (en) 2021-01-28
EP3802812A1 (en) 2021-04-14
EP3802812A4 (en) 2022-03-30
US20210047654A1 (en) 2021-02-18
US20200071718A1 (en) 2020-03-05
SG11202012004SA (en) 2021-01-28
CA3102779A1 (en) 2019-12-12
JP2021526858A (ja) 2021-10-11
CN112930395A (zh) 2021-06-08
US20200123569A1 (en) 2020-04-23
WO2019236982A1 (en) 2019-12-12

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