JP7720623B6 - 遺伝子操作されたａｄａｒのリクルートを介したｒｎａ及びｄｎａ塩基の編集 - Google Patents

遺伝子操作されたａｄａｒのリクルートを介したｒｎａ及びｄｎａ塩基の編集

Info

Publication number
JP7720623B6
JP7720623B6 JP2021511542A JP2021511542A JP7720623B6 JP 7720623 B6 JP7720623 B6 JP 7720623B6 JP 2021511542 A JP2021511542 A JP 2021511542A JP 2021511542 A JP2021511542 A JP 2021511542A JP 7720623 B6 JP7720623 B6 JP 7720623B6
Authority
JP
Japan
Prior art keywords
rna
cases
editing
domain
sequence
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.)
Active
Application number
JP2021511542A
Other languages
English (en)
Japanese (ja)
Other versions
JP7720623B2 (ja
JP2021536244A5 (https=
JP2021536244A (ja
Inventor
マリ、プラシャント
カプレカー、ドゥルヴァ
メルッツィ、ダリオ
チェン、ゴンハオ
エム フォード、カイル
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 JP2021536244A publication Critical patent/JP2021536244A/ja
Publication of JP2021536244A5 publication Critical patent/JP2021536244A5/ja
Publication of JP7720623B2 publication Critical patent/JP7720623B2/ja
Application granted granted Critical
Publication of JP7720623B6 publication Critical patent/JP7720623B6/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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
    • 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
    • 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
    • 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
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/16—Aptamers
    • 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
    • C12N2310/20—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
    • 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
    • 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
    • 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
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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
    • C12N2320/33—Alteration of splicing
    • 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
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen
    • 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
    • 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
    • C12N2750/14311—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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • 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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (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)
JP2021511542A 2018-09-06 2019-09-06 遺伝子操作されたａｄａｒのリクルートを介したｒｎａ及びｄｎａ塩基の編集 Active JP7720623B6 (ja)

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 (4)

Publication Number Publication Date
JP2021536244A JP2021536244A (ja) 2021-12-27
JP2021536244A5 JP2021536244A5 (https=) 2022-10-19
JP7720623B2 JP7720623B2 (ja) 2025-08-08
JP7720623B6 true JP7720623B6 (ja) 2025-09-18

Family

ID=69723301

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021511542A Active JP7720623B6 (ja) 2018-09-06 2019-09-06 遺伝子操作されたａｄａｒのリクルートを介したｒｎａ及びｄｎａ塩基の編集

Country Status (8)

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

Families Citing this family (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110612353A (zh) * 2017-03-03 2019-12-24 加利福尼亚大学董事会 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向
WO2019104094A2 (en) 2017-11-21 2019-05-31 The Regents Of The University Of California Fusion proteins and methods for site-directed genome editing
EP3956449B1 (en) 2019-04-15 2026-02-25 EdiGene Therapeutics (Beijing) Inc. Methods and compositions for editing rnas
EP3997229A4 (en) * 2019-07-12 2024-07-03 Peking University TARGETED RNA EDITING BY HARNESSING ENDOGENOUS ADAR USING MODIFIED RNA
WO2021113264A1 (en) * 2019-12-02 2021-06-10 The Regents Of The University Of California Engineering circular guide rnas
WO2021113390A1 (en) * 2019-12-02 2021-06-10 Shape Therapeutics Inc. Compositions for treatment of diseases
AU2020395113A1 (en) 2019-12-02 2022-06-09 Shape Therapeutics Inc. Therapeutic editing
US20230059753A1 (en) * 2019-12-09 2023-02-23 Astellas Pharma Inc. Antisense guide rna with added functional region for editing target rna
CN115777020A (zh) * 2020-04-22 2023-03-10 塑造治疗公司 使用snrna组分的组合物和方法
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
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
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
US20230272379A1 (en) * 2020-07-06 2023-08-31 Edigene Therapeutics (Beijing ) Inc. Improved rna editing method
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
WO2022046667A1 (en) * 2020-08-24 2022-03-03 Wave Life Sciences Ltd. Cells and non-human animals engineered to express adar1 and uses thereof
WO2022078569A1 (en) * 2020-10-12 2022-04-21 Eberhard Karls Universität Tübingen Artificial nucleic acids for rna editing
JP2024500279A (ja) * 2020-11-11 2024-01-09 シェイプ セラピューティクス インコーポレイテッド Ｒｎａ編集組成物及び使用方法
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
WO2023278436A1 (en) * 2021-06-29 2023-01-05 Shape Therapeutics Inc. Engineered guide rnas and polynucleotides
EP4388089A4 (en) * 2021-08-18 2025-01-15 Peking University Engineered adar-recruiting rnas and methods of use thereof
AU2022337146A1 (en) 2021-09-03 2024-03-14 Tacit Therapeutics, Inc. Rna editing via recruitment of spliceosome components
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
WO2023196853A1 (en) * 2022-04-05 2023-10-12 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
WO2026080897A1 (en) * 2024-10-11 2026-04-16 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of chronic pain

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2017537618A (ja) 2014-12-17 2017-12-21 プロキューアール・セラピューティクス・セカンド・ベスローテン・フェンノートシャップＰｒｏＱＲ Ｔｈｅｒａｐｅｕｔｉｃｓ ＩＩ Ｂ．Ｖ． 標的化ｒｎａ編集
WO2017220751A1 (en) 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Single-stranded rna-editing oligonucleotides

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
FR2764387B1 (fr) * 1997-06-05 1999-07-23 Centre Nat Rech Scient Utilisation d'une proteine fluorescente pour la detection d'interactions entre une proteine cible et son ligand
MX367100B (es) * 2012-02-17 2019-08-05 The Children´S Hospital Of Philadelphia Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos.
CA3005245A1 (en) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of alagille syndrome
CN109642231A (zh) * 2016-06-17 2019-04-16 博德研究所 Vi型crispr直向同源物和系统
WO2018208998A1 (en) * 2017-05-10 2018-11-15 The Regents Of The University Of California Directed editing of cellular rna via nuclear delivery of crispr/cas9

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2017537618A (ja) 2014-12-17 2017-12-21 プロキューアール・セラピューティクス・セカンド・ベスローテン・フェンノートシャップＰｒｏＱＲ Ｔｈｅｒａｐｅｕｔｉｃｓ ＩＩ Ｂ．Ｖ． 標的化ｒｎａ編集
WO2017220751A1 (en) 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Single-stranded rna-editing oligonucleotides

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
Nature Biotechnology，2018年07月15日，vol.37, no.9，p.1059-1069
PNAS USA，2013年，vol.110, no.45，p.18285-18290
Scientific Reports，2017年02月02日，vol.7，41478

Also Published As

Publication number Publication date
CA3110998A1 (en) 2020-03-12
JP7720623B2 (ja) 2025-08-08
EP4332224A2 (en) 2024-03-06
EP3847255A4 (en) 2024-01-10
EP3847255A1 (en) 2021-07-14
AU2019336793A1 (en) 2021-04-08
CN119193702A (zh) 2024-12-27
AU2019336793B2 (en) 2024-11-21
US20260092292A1 (en) 2026-04-02
GB202104889D0 (en) 2021-05-19
US20220010333A1 (en) 2022-01-13
EP4332224A3 (en) 2024-06-05
WO2020051555A1 (en) 2020-03-12
JP2021536244A (ja) 2021-12-27
GB2590880A (en) 2021-07-07
CN112996912A (zh) 2021-06-18

Similar Documents

Publication Publication Date Title
JP7720623B6 (ja) 遺伝子操作されたａｄａｒのリクルートを介したｒｎａ及びｄｎａ塩基の編集
EP4100032B1 (en) Gene editing methods for treating spinal muscular atrophy
US20240392303A1 (en) RNA TARGETING OF MUTATIONS VIA SUPPRESSOR tRNAs AND DEAMINASES
KR20200074283A (ko) 표적 세포 내에서 치료적 단백질의 표적화된 생산을 위한 시스템 및 방법
US20250270543A1 (en) Programmable rna editing in vivo via recruitment of endogenous adars
JP7755272B2 (ja) 操作されたａｄａｒ動員ｒｎａ及びその使用方法
US20230174958A1 (en) Crispr-inhibition for facioscapulohumeral muscular dystrophy
US20230313231A1 (en) Rna and dna base editing via engineered adar
CN118043461A (zh) 方法
US20260091141A1 (en) Gene editing methods, systems, and compositions for treating spinal muscular atrophy
WO2022239863A1 (ja) アンチセンスオリゴマー
KR20250158061A (ko) Pah-조절 시스템 및 방법
HK40119835A (zh) 用於靶向pcsk9的组合物和方法

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220906

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20220906

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220919

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220922

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20230908

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20231122

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240206

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20240510

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240809

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20241105

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20241209

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250307

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20250609

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20250708

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20250722

R150 Certificate of patent or registration of utility model

Ref document number: 7720623

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150