CA3159799A1 - Engineering circular guide rnas - Google Patents

Engineering circular guide rnas Download PDF

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Publication number
CA3159799A1
CA3159799A1 CA3159799A CA3159799A CA3159799A1 CA 3159799 A1 CA3159799 A1 CA 3159799A1 CA 3159799 A CA3159799 A CA 3159799A CA 3159799 A CA3159799 A CA 3159799A CA 3159799 A1 CA3159799 A1 CA 3159799A1
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CA
Canada
Prior art keywords
rna
engineered
sequence
guide rna
engineered guide
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
CA3159799A
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English (en)
French (fr)
Inventor
Prashant MALI
Dhruva KATREKAR
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of California San Diego UCSD
Original Assignee
University of California San Diego UCSD
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by University of California San Diego UCSD filed Critical University of California San Diego UCSD
Publication of CA3159799A1 publication Critical patent/CA3159799A1/en
Pending legal-status Critical Current

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/532—Closed or circular
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • 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)
CA3159799A 2019-12-02 2020-12-01 Engineering circular guide rnas Pending CA3159799A1 (en)

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

Publications (1)

Publication Number Publication Date
CA3159799A1 true CA3159799A1 (en) 2021-06-10

Family

ID=76221931

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3159799A Pending CA3159799A1 (en) 2019-12-02 2020-12-01 Engineering circular guide rnas

Country Status (7)

Country Link
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

Family Cites Families (9)

* Cited by examiner, † Cited by third party
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
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

Also Published As

Publication number Publication date
JP2023504048A (ja) 2023-02-01
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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