CA3110998A1 - Rna and dna base editing via engneered adar recruitment - Google Patents

Rna and dna base editing via engneered adar recruitment Download PDF

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Publication number
CA3110998A1
CA3110998A1 CA3110998A CA3110998A CA3110998A1 CA 3110998 A1 CA3110998 A1 CA 3110998A1 CA 3110998 A CA3110998 A CA 3110998A CA 3110998 A CA3110998 A CA 3110998A CA 3110998 A1 CA3110998 A1 CA 3110998A1
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domain
nucleic acid
rna
vector
sequence
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CA3110998A
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English (en)
French (fr)
Inventor
Prashant MALI
Dhruva KATREKAR
Dario Meluzzi
Genghao CHEN
Kyle M. FORD
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University of California, San Diego
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University of California, San Diego
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Publication of CA3110998A1 publication Critical patent/CA3110998A1/en
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    • C—CHEMISTRY; METALLURGY
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    • 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
    • C12N15/63—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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/16—Aptamers
    • C—CHEMISTRY; METALLURGY
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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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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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
    • C12N2310/531—Stem-loop; Hairpin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen
    • C—CHEMISTRY; METALLURGY
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    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14311—Parvovirus, e.g. minute virus of mice
    • C12N2750/14341—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Heterocyclic Carbon Compounds Containing A Hetero Ring Having Nitrogen And Oxygen As The Only Ring Hetero Atoms (AREA)
  • Nitrogen And Oxygen Or Sulfur-Condensed Heterocyclic Ring Systems (AREA)
CA3110998A 2018-09-06 2019-09-06 Rna and dna base editing via engneered adar recruitment Pending CA3110998A1 (en)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201862728007P 2018-09-06 2018-09-06
US62/728,007 2018-09-06
US201862766433P 2018-10-17 2018-10-17
US62/766,433 2018-10-17
US201862773150P 2018-11-29 2018-11-29
US201862773146P 2018-11-29 2018-11-29
US62/773,146 2018-11-29
US62/773,150 2018-11-29
US201862780241P 2018-12-15 2018-12-15
US62/780,241 2018-12-15
PCT/US2019/050095 WO2020051555A1 (en) 2018-09-06 2019-09-06 Rna and dna base editing via engneered adar recruitment

Publications (1)

Publication Number Publication Date
CA3110998A1 true CA3110998A1 (en) 2020-03-12

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CA3110998A Pending CA3110998A1 (en) 2018-09-06 2019-09-06 Rna and dna base editing via engneered adar recruitment

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US (2) US20220010333A1 (enExample)
EP (2) EP4332224A3 (enExample)
JP (1) JP7720623B6 (enExample)
CN (2) CN119193702A (enExample)
AU (1) AU2019336793B2 (enExample)
CA (1) CA3110998A1 (enExample)
GB (1) GB2590880A (enExample)
WO (1) WO2020051555A1 (enExample)

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WO2019104094A2 (en) 2017-11-21 2019-05-31 The Regents Of The University Of California Fusion proteins and methods for site-directed genome editing
CA3136735A1 (en) 2019-04-15 2020-10-22 Edigene Inc. Methods and compositions for editing rnas
AU2020313143B2 (en) * 2019-07-12 2024-04-04 Peking University Targeted RNA editing by leveraging endogenous adar using engineered RNAs
WO2021113390A1 (en) * 2019-12-02 2021-06-10 Shape Therapeutics Inc. Compositions for treatment of diseases
CA3159799A1 (en) * 2019-12-02 2021-06-10 Prashant MALI Engineering circular guide rnas
US11827880B2 (en) 2019-12-02 2023-11-28 Shape Therapeutics Inc. Therapeutic editing
IL293671A (en) * 2019-12-09 2022-08-01 Astellas Pharma Inc Antisense guide RNA with an additional functional region for target RNA editing
CA3173012A1 (en) * 2020-04-22 2021-10-28 Susan BYRNE Compositions and methods using snrna components
JP2023529316A (ja) * 2020-05-26 2023-07-10 シェイプ セラピューティクス インコーポレイテッド ゲノム編集のための組成物及び方法
EP4158019A1 (en) * 2020-05-26 2023-04-05 Shape Therapeutics Inc. Methods and compositions relating to engineered guide systems for adenosine deaminase acting on rna editing
EP4158017A1 (en) * 2020-05-26 2023-04-05 Shape Therapeutics Inc. Engineered circular polynucleotides
KR20230035362A (ko) * 2020-07-06 2023-03-13 에디진 테라퓨틱스 (베이징) 인크. 개선된 rna 편집 방법
CN116209762A (zh) * 2020-07-30 2023-06-02 阿达尔克斯制药有限公司 Adar依赖性编辑组合物及其使用方法
CN115989041A (zh) * 2020-08-24 2023-04-18 波涛生命科学有限公司 经工程化为表达adar1的细胞和非人动物及其用途
WO2022078569A1 (en) * 2020-10-12 2022-04-21 Eberhard Karls Universität Tübingen Artificial nucleic acids for rna editing
US20230399635A1 (en) * 2020-11-11 2023-12-14 Shape Therapeutics Inc. RNA-Editing Compositions and Methods of Use
CN116648508A (zh) * 2020-11-11 2023-08-25 塑造治疗公司 Rna编辑组合物和使用方法
EP4256050A4 (en) * 2020-12-01 2025-01-22 Shape Therapeutics Inc. High throughput rna-editing screening methods
US20240150754A1 (en) * 2020-12-25 2024-05-09 Astellas Pharma Inc. Guide rna for editing polyadenylation signal sequence of target rna
CA3220338A1 (en) * 2021-05-25 2022-12-01 Yiannis SAVVA Engineered guide rnas and polynucleotides
US20240279656A1 (en) * 2021-06-29 2024-08-22 Shape Therapeutics Inc. Engineered Guide RNAs and Polynucleotides
TW202321451A (zh) * 2021-08-18 2023-06-01 北京大學 工程化的 adar 招募 rnas 及其使用方法
JP2024533163A (ja) 2021-09-03 2024-09-12 タシト・セラピューティクス・インコーポレーテッド スプライソソーム構成要素のリクルートを介したｒｎａ編集
JP2023040852A (ja) * 2021-09-10 2023-03-23 隆光 矢野 －１フレームシフトを誘導するための１本鎖核酸分子及び組成物
US20230167441A1 (en) * 2021-11-30 2023-06-01 Minghong Zhong Segmented Nucleic Acids
US20250115908A1 (en) * 2022-02-07 2025-04-10 The Regents Of The University Of California Method of interfering with repetitive rna
US20250215426A1 (en) * 2022-04-05 2025-07-03 Astellas Gene Therapies, Inc. Compositions and methods for the treatment of muscular dystrophies
CN116732039B (zh) * 2023-08-03 2023-11-14 呈诺再生医学科技(北京)有限公司 一种促进rna序列在细胞内直接环化翻译的序列组合及其应用
CN117210435A (zh) * 2023-08-29 2023-12-12 苏州湃芮生物科技有限公司 一种用于调控rna甲基化修饰的编辑系统及其应用
CN121059628A (zh) * 2024-06-03 2025-12-05 时夕(广州)生物科技有限公司 调控RNA剪接的向导agRNA
WO2026061377A1 (zh) * 2024-09-18 2026-03-26 时夕(广州)生物科技有限公司 Adar向导rna及其应用
WO2026080897A1 (en) * 2024-10-11 2026-04-16 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of chronic pain

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EP3847255A4 (en) 2024-01-10
EP4332224A2 (en) 2024-03-06
US20260092292A1 (en) 2026-04-02
CN112996912A (zh) 2021-06-18
JP7720623B6 (ja) 2025-09-18
EP3847255A1 (en) 2021-07-14
JP2021536244A (ja) 2021-12-27
GB2590880A (en) 2021-07-07
GB202104889D0 (en) 2021-05-19
WO2020051555A1 (en) 2020-03-12
JP7720623B2 (ja) 2025-08-08
AU2019336793A1 (en) 2021-04-08
CN119193702A (zh) 2024-12-27
AU2019336793B2 (en) 2024-11-21
EP4332224A3 (en) 2024-06-05
US20220010333A1 (en) 2022-01-13

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