CN114174510A - 添加了功能性碱基序列的靶标编辑指导rna - Google Patents

添加了功能性碱基序列的靶标编辑指导rna Download PDF

Info

Publication number
CN114174510A
CN114174510A CN202080054392.3A CN202080054392A CN114174510A CN 114174510 A CN114174510 A CN 114174510A CN 202080054392 A CN202080054392 A CN 202080054392A CN 114174510 A CN114174510 A CN 114174510A
Authority
CN
China
Prior art keywords
sequence
base sequence
guide rna
adar
rna
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
CN202080054392.3A
Other languages
English (en)
Chinese (zh)
Inventor
吉见英治
森家由香里
万田茉莉子
福田将虎
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.)
Astellas Pharma Inc
Fukuoka University
Original Assignee
Astellas Pharma Inc
Fukuoka University
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 Astellas Pharma Inc, Fukuoka University filed Critical Astellas Pharma Inc
Publication of CN114174510A publication Critical patent/CN114174510A/zh
Pending legal-status Critical Current

Links

Images

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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • 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
    • 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
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0684—Cells of the urinary tract or kidneys
    • C12N5/0686—Kidney cells
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/10—Cells modified by introduction of foreign genetic material
    • 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
    • C12Y—ENZYMES
    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • 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
    • C12N2310/50—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
    • C12N2510/00—Genetically modified cells
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Cell Biology (AREA)
  • Urology & Nephrology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CN202080054392.3A 2019-08-01 2020-07-31 添加了功能性碱基序列的靶标编辑指导rna Pending CN114174510A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2019-141875 2019-08-01
JP2019141875 2019-08-01
PCT/JP2020/029386 WO2021020550A1 (ja) 2019-08-01 2020-07-31 機能性塩基配列を付加した標的編集ガイドｒｎａ

Publications (1)

Publication Number Publication Date
CN114174510A true CN114174510A (zh) 2022-03-11

Family

ID=74229702

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202080054392.3A Pending CN114174510A (zh) 2019-08-01 2020-07-31 添加了功能性碱基序列的靶标编辑指导rna

Country Status (5)

Country Link
US (1) US20220275359A1 (https=)
EP (1) EP4008784A1 (https=)
JP (1) JPWO2021020550A1 (https=)
CN (1) CN114174510A (https=)
WO (1) WO2021020550A1 (https=)

Families Citing this family (33)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017220751A1 (en) 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Single-stranded rna-editing oligonucleotides
CN110352244B (zh) 2016-09-01 2023-03-21 ProQR治疗上市公司Ⅱ 化学修饰的编辑rna的单链寡核苷酸
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
AU2020395113A1 (en) 2019-12-02 2022-06-09 Shape Therapeutics Inc. Therapeutic editing
US20240247258A1 (en) * 2020-03-12 2024-07-25 Frest Inc. Oligonucleotide and target rna site-specific editing method
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
US20240150754A1 (en) 2020-12-25 2024-05-09 Astellas Pharma Inc. Guide rna for editing polyadenylation signal sequence of target rna
AU2022375788A1 (en) * 2021-10-27 2024-05-23 Shape Therapeutics Inc. Engineered rnas
US20250115908A1 (en) * 2022-02-07 2025-04-10 The Regents Of The University Of California Method of interfering with repetitive rna
EP4479535A1 (en) 2022-02-14 2024-12-25 ProQR Therapeutics II B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
JP2025524566A (ja) 2022-07-15 2025-07-30 プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ Ａｄａｒ媒介ｒｎａ編集のためのオリゴヌクレオチドおよびその使用
US20260027237A1 (en) 2022-07-15 2026-01-29 Proqr Therapeutics Ii B.V. Chemically modified oligonucleotides for adar-mediated rna editing
AU2023364059A1 (en) * 2022-10-18 2025-05-08 Regeneron Pharmaceuticals, Inc. Compositions and methods comprising programmable snrnas for rna editing
GB202215614D0 (en) 2022-10-21 2022-12-07 Proqr Therapeutics Ii Bv Heteroduplex rna editing oligonucleotide complexes
WO2024110565A1 (en) 2022-11-24 2024-05-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of hereditary hfe-hemochromatosis
GB202218090D0 (en) 2022-12-01 2023-01-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency
AU2023390632A1 (en) 2022-12-09 2025-07-10 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of cardiovascular disease
GB202300865D0 (en) 2023-01-20 2023-03-08 Proqr Therapeutics Ii Bv Delivery of oligonucleotides
WO2024175550A1 (en) 2023-02-20 2024-08-29 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease
EP4689110A1 (en) 2023-03-24 2026-02-11 ProQR Therapeutics II B.V. Antisense oligonucleotides for the treatment of neurological disorders
GB202304363D0 (en) 2023-03-24 2023-05-10 Proqr Therapeutics Ii Bv Chemically modified antisense oligonucleotides for use in RNA editing
EP4688143A1 (en) 2023-03-27 2026-02-11 ProQR Therapeutics II B.V. Antisense oligonucleotides for the treatment of liver disease
WO2024226860A2 (en) * 2023-04-26 2024-10-31 Shape Therapeutics Inc. Small nuclear rna processing hairpins for small rna payloads
TW202516003A (zh) 2023-06-16 2025-04-16 荷蘭商Ｐｒｏｑｒ治療上市公司Ｉｉ 用於治療神經退化性疾病之反義寡核苷酸
WO2025051946A1 (en) 2023-09-07 2025-03-13 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of metabolic disorders
WO2025104239A1 (en) 2023-11-16 2025-05-22 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of classic galactosemia
WO2025132708A1 (en) 2023-12-20 2025-06-26 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of huntington's disease
GB202404661D0 (en) 2024-04-02 2024-05-15 Proqr Therapeutics Ii Bv Antisense oligoncleotides for the treatment of liver disease
WO2025224230A1 (en) 2024-04-25 2025-10-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of fatty liver disease
GB202410081D0 (en) 2024-07-11 2024-08-28 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of cardiovascular disease
WO2026022136A1 (en) 2024-07-23 2026-01-29 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of metabolic disorders
WO2026060374A2 (en) 2024-09-16 2026-03-19 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of neurological disorders
WO2026068781A1 (en) 2024-09-30 2026-04-02 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of liver disease

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN106062197A (zh) * 2013-06-17 2016-10-26 布罗德研究所有限公司 用于序列操纵的串联指导系统、方法和组合物的递送、工程化和优化
CN108291218A (zh) * 2015-07-15 2018-07-17 新泽西鲁特格斯州立大学 核酸酶非依赖性靶向基因编辑平台及其用途
US20190093098A1 (en) * 2015-09-26 2019-03-28 Eberhard Karls Universität Tübingen Methods and substances for directed rna editing
JP2019097555A (ja) * 2017-12-06 2019-06-24 学校法人福岡大学 オリゴヌクレオチド、その製造方法及び標的ｒｎａの部位特異的編集方法

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030148519A1 (en) * 2001-11-14 2003-08-07 Engelke David R. Intracellular expression and delivery of siRNAs in mammalian cells
EP3234134B1 (en) 2014-12-17 2020-05-27 ProQR Therapeutics II B.V. Targeted rna editing
EP3323890A4 (en) 2015-07-14 2019-01-30 Fukuoka University METHOD OF INDUSTRYING SITE-SPECIFIC RNA MUTATIONS, TARGETED EDITING GUIDE RNA USED IN THE METHOD AND TARGET RNA TARGET EDITING GUIDE RNA COMPLEX
WO2017220751A1 (en) 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Single-stranded rna-editing oligonucleotides
CN110352244B (zh) 2016-09-01 2023-03-21 ProQR治疗上市公司Ⅱ 化学修饰的编辑rna的单链寡核苷酸
EP3571300A1 (en) 2017-01-19 2019-11-27 ProQR Therapeutics II B.V. Oligonucleotide complexes for use in rna editing
WO2019111957A1 (ja) 2017-12-06 2019-06-13 学校法人福岡大学 オリゴヌクレオチド、その製造方法及び標的ｒｎａの部位特異的編集方法

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN106062197A (zh) * 2013-06-17 2016-10-26 布罗德研究所有限公司 用于序列操纵的串联指导系统、方法和组合物的递送、工程化和优化
CN108291218A (zh) * 2015-07-15 2018-07-17 新泽西鲁特格斯州立大学 核酸酶非依赖性靶向基因编辑平台及其用途
US20190093098A1 (en) * 2015-09-26 2019-03-28 Eberhard Karls Universität Tübingen Methods and substances for directed rna editing
JP2019097555A (ja) * 2017-12-06 2019-06-24 学校法人福岡大学 オリゴヌクレオチド、その製造方法及び標的ｒｎａの部位特異的編集方法

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
GOOD, PD等: ""Expression of small, therapeutic RNAs in human cell nuclei"", 《GENE THERAPY》, vol. 4, no. 1, pages 3 - 4 *
NAHAR, SMITA等: ""A G-quadruplex motif at the 3\' end of sgRNAs improves CRISPR-Cas9 based genome editing efficiency"", 《CHEMICAL COMMUNICATION》, vol. 54, no. 19, pages 2, XP055789487, DOI: 10.1039/C7CC08893K *

Also Published As

Publication number Publication date
WO2021020550A1 (ja) 2021-02-04
JPWO2021020550A1 (https=) 2021-02-04
EP4008784A1 (en) 2022-06-08
US20220275359A1 (en) 2022-09-01

Similar Documents

Publication Publication Date Title
CN114174510A (zh) 添加了功能性碱基序列的靶标编辑指导rna
JP7754721B2 (ja) 標的ｒｎａを編集するための機能性領域を付加したアンチセンス型ガイドｒｎａ
US11649454B2 (en) Single-stranded RNA-editing oligonucleotides
US11939580B2 (en) Construct of self-circularization RNA
Montiel-Gonzalez et al. Current strategies for site-directed RNA editing using ADARs
US20240425902A1 (en) Methods of purifying dna for gene synthesis
CA2968336C (en) Construct for site directed editing of an adenosine nucleotide in target rna
KR101900962B1 (ko) 섬유아세포 성장 인자 21 (ｆｇｆ21)에 대한 자연 안티센스 전사체의 저해에 의한 섬유아세포 성장 인자 21 (ｆｇｆ21) 관련된 질환의 치료
CN117242184A (zh) 向导RNA设计及用于V型Cas系统的复合物
US12467045B2 (en) Tuning CRISPR/Cas9 activity with chemically modified nucleotide substitutions
JP7432521B2 (ja) 新規小分子活性化ｒｎａ
JPH08502408A (ja) 特定のｒｎａ鎖の切断方法
US11643658B2 (en) Oligonucleotides, manufacturing method for same, and target RNA site-specific editing method
EP4269585A1 (en) Guide rna for editing polyadenylation signal sequence of target rna
CN116897047A (zh) 一种有效沉默细胞中的基因表达的生物稳定的XNAzyme
KR20220122727A (ko) Rna의 표적 편집을 위한 신규한 방법
CN117210435A (zh) 一种用于调控rna甲基化修饰的编辑系统及其应用
HK40076284A (en) Antisense guide rna with added functional region for editing target rna
EP3964582A1 (en) Oligomeric nucleic acid molecule, and application thereof in acute intermittent porphyria treatment
KR20260029512A (ko) 안정성이 개선된 삼중나선 구조의 핵산 분자 및 이의 용도
JP2025071533A (ja) 新型誘導細胞質インビトロ転写様組成物およびその関連ワクチン医薬設計
HK40078456A (en) Use of sbds inhibitors for treating hepatitis b virus infection

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
WD01 Invention patent application deemed withdrawn after publication
WD01 Invention patent application deemed withdrawn after publication

Application publication date: 20220311