CN115210366A - 工程化环状指导rna - Google Patents

工程化环状指导rna Download PDF

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Publication number
CN115210366A
CN115210366A CN202080094276.4A CN202080094276A CN115210366A CN 115210366 A CN115210366 A CN 115210366A CN 202080094276 A CN202080094276 A CN 202080094276A CN 115210366 A CN115210366 A CN 115210366A
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rna
engineered
sequence
guide rna
engineered guide
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CN202080094276.4A
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Chinese (zh)
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P·马里
D·卡特里卡
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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
    • C12N2310/35—Nature of the modification
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    • C12N2310/3519—Fusion with another nucleic acid
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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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    • 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)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CN202080094276.4A 2019-12-02 2020-12-01 工程化环状指导rna Pending CN115210366A (zh)

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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Publication Number Publication Date
CN115210366A true CN115210366A (zh) 2022-10-18

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CN202080094276.4A Pending CN115210366A (zh) 2019-12-02 2020-12-01 工程化环状指导rna

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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 (3)

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WO2018237372A1 (en) * 2017-06-23 2018-12-27 Cornell University RNA MOLECULES, METHODS FOR PRODUCING CIRCULAR RNA, AND METHODS OF TREATMENT
WO2019005884A1 (en) * 2017-06-26 2019-01-03 The Broad Institute, Inc. CRISPR / CAS-ADENINE DEAMINASE COMPOSITIONS, SYSTEMS AND METHODS FOR TARGETED NUCLEIC ACID EDITION
CN114269919A (zh) * 2019-07-12 2022-04-01 北京大学 采用工程化rna利用内源adar进行靶向的rna编辑

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Publication number Priority date Publication date Assignee Title
DK2925866T3 (en) * 2012-11-30 2018-10-29 Univ Aarhus CIRCULAR RNA FOR INHIBITING MICRO-RNA
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
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
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 (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018237372A1 (en) * 2017-06-23 2018-12-27 Cornell University RNA MOLECULES, METHODS FOR PRODUCING CIRCULAR RNA, AND METHODS OF TREATMENT
WO2019005884A1 (en) * 2017-06-26 2019-01-03 The Broad Institute, Inc. CRISPR / CAS-ADENINE DEAMINASE COMPOSITIONS, SYSTEMS AND METHODS FOR TARGETED NUCLEIC ACID EDITION
CN114269919A (zh) * 2019-07-12 2022-04-01 北京大学 采用工程化rna利用内源adar进行靶向的rna编辑

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
KATREKAR D等: "In vivo RNA editing of point mutations via RNA-guided adenosine deaminases. Nat Methods", NAT METHODS, vol. 16, no. 3, 31 March 2019 (2019-03-31), pages 239 - 242 *
MARTIN WEGNER等: "Circular synthesized CRISPR/Cas gRNAs for functional interrogations in the coding and noncoding genome", ELIFE, 6 March 2019 (2019-03-06), pages 1 - 31 *
QU L等: "Programmable RNA editing by recruiting endogenous ADAR using engineered RNAs", NAT BIOTECHNOL, vol. 37, no. 9, 30 September 2019 (2019-09-30), pages 1059 - 1069, XP036888288, DOI: 10.1038/s41587-019-0178-z *

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CA3159799A1 (en) 2021-06-10
JP2023504048A (ja) 2023-02-01
EP4069830A4 (en) 2024-10-02
WO2021113264A1 (en) 2021-06-10
EP4069830A1 (en) 2022-10-12
US20230036370A1 (en) 2023-02-02
AU2020395111A1 (en) 2022-06-02

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Application publication date: 20221018