JP2023504048A - 環状ガイドｒｎａの遺伝子操作 - Google Patents

環状ガイドｒｎａの遺伝子操作 Download PDF

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JP2023504048A
JP2023504048A JP2022530897A JP2022530897A JP2023504048A JP 2023504048 A JP2023504048 A JP 2023504048A JP 2022530897 A JP2022530897 A JP 2022530897A JP 2022530897 A JP2022530897 A JP 2022530897A JP 2023504048 A JP2023504048 A JP 2023504048A
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rna
artificial
guide rna
sequence
polynucleotide
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JP2023504048A5 (enExample
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マリ、プラシャント
カトレカール、ドルバ
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University of California San Diego UCSD
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University of California San Diego UCSD
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    • 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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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
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    • C12N2310/532—Closed or circular
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    • C12N2320/00—Applications; Uses
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
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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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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2022530897A 2019-12-02 2020-12-01 環状ガイドｒｎａの遺伝子操作 Pending JP2023504048A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962942725P 2019-12-02 2019-12-02
US62/942,725 2019-12-02
US202063112492P 2020-11-11 2020-11-11
US63/112,492 2020-11-11
PCT/US2020/062748 WO2021113264A1 (en) 2019-12-02 2020-12-01 Engineering circular guide rnas

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JP2023504048A true JP2023504048A (ja) 2023-02-01
JP2023504048A5 JP2023504048A5 (enExample) 2023-12-04

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US (1) US20230036370A1 (enExample)
EP (1) EP4069830A4 (enExample)
JP (1) JP2023504048A (enExample)
CN (1) CN115210366A (enExample)
AU (1) AU2020395111A1 (enExample)
CA (1) CA3159799A1 (enExample)
WO (1) WO2021113264A1 (enExample)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20230055682A1 (en) * 2019-12-03 2023-02-23 Beam Therapeutics lnc. Synthetic guide rna, compositions, methods, and uses thereof
US20230059753A1 (en) * 2019-12-09 2023-02-23 Astellas Pharma Inc. Antisense guide rna with added functional region for editing target rna
JP2023528039A (ja) * 2020-05-26 2023-07-03 シェイプ セラピューティクス インコーポレイテッド 改変環状ポリヌクレオチド
WO2022147573A1 (en) * 2021-01-04 2022-07-07 The Regents Of The University Of California Programmable rna editing in vivo via recruitment of endogenous adars
JP2024504608A (ja) * 2021-01-12 2024-02-01 北京大学 遺伝子操作ｒｎａを使用した内因性ａｄａｒの活用による標的化ｒｎａの編集
EP4423274A2 (en) * 2021-10-26 2024-09-04 Shape Therapeutics Inc. Rna-editing compositions and methods of use
EP4667570A1 (en) * 2024-06-20 2025-12-24 Johannes Gutenberg-Universität Mainz Nucleic acid molecule complex for target pseudouridylation in mammalian cells

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014082644A1 (en) * 2012-11-30 2014-06-05 WULFF, Peter, Samuel Circular rna for inhibition of microrna

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CN120574876A (zh) * 2013-08-22 2025-09-02 纳幕尔杜邦公司 使用向导rna/cas内切核酸酶系统的植物基因组修饰及其使用方法
WO2018237372A1 (en) * 2017-06-23 2018-12-27 Cornell University RNA MOLECULES, METHODS FOR PRODUCING CIRCULAR RNA, AND METHODS OF TREATMENT
KR102761791B1 (ko) * 2017-06-26 2025-02-05 더 브로드 인스티튜트, 인코퍼레이티드 표적화된 핵산 편집을 위한 crispr/cas-아데닌 데아미나아제 기반 조성물, 시스템 및 방법
US20200362334A1 (en) * 2017-12-07 2020-11-19 The Broad Institute, Inc. High-throughput methods for identifying gene interactions and networks
CN111819185A (zh) * 2017-12-15 2020-10-23 旗舰创业创新第六有限责任公司 包含环状多核糖核苷酸的组合物及其用途
US20220010333A1 (en) * 2018-09-06 2022-01-13 The Regents Of The University Of California Rna and dna base editing via engineered adar recruitment
EP3997229A4 (en) * 2019-07-12 2024-07-03 Peking University TARGETED RNA EDITING BY HARNESSING ENDOGENOUS ADAR USING MODIFIED RNA
JP2023528039A (ja) * 2020-05-26 2023-07-03 シェイプ セラピューティクス インコーポレイテッド 改変環状ポリヌクレオチド

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014082644A1 (en) * 2012-11-30 2014-06-05 WULFF, Peter, Samuel Circular rna for inhibition of microrna

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CA3159799A1 (en) 2021-06-10
EP4069830A4 (en) 2024-10-02
WO2021113264A1 (en) 2021-06-10
CN115210366A (zh) 2022-10-18
EP4069830A1 (en) 2022-10-12
US20230036370A1 (en) 2023-02-02
AU2020395111A1 (en) 2022-06-02

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