BR112022011277A2 - Rna guia anti-sentido com região funcional adicionada para edição de rna alvo - Google Patents

Rna guia anti-sentido com região funcional adicionada para edição de rna alvo

Info

Publication number
BR112022011277A2
BR112022011277A2 BR112022011277A BR112022011277A BR112022011277A2 BR 112022011277 A2 BR112022011277 A2 BR 112022011277A2 BR 112022011277 A BR112022011277 A BR 112022011277A BR 112022011277 A BR112022011277 A BR 112022011277A BR 112022011277 A2 BR112022011277 A2 BR 112022011277A2
Authority
BR
Brazil
Prior art keywords
rna
functional region
target rna
antisense
editing
Prior art date
Application number
BR112022011277A
Other languages
English (en)
Inventor
Yoshimi Eiji
Moriya Yukari
Manda Mariko
Gotoh Takayasu
Arai Takatomo
Original Assignee
Astellas Pharma Inc
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 filed Critical Astellas Pharma Inc
Publication of BR112022011277A2 publication Critical patent/BR112022011277A2/pt

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/50Hydrolases (3) acting on carbon-nitrogen bonds, other than peptide bonds (3.5), e.g. asparaginase
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/74Vectors or expression systems specially adapted for prokaryotic hosts other than E. coli, e.g. Lactobacillus, Micromonospora
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/80Vectors or expression systems specially adapted for eukaryotic hosts for fungi
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/80Vectors or expression systems specially adapted for eukaryotic hosts for fungi
    • C12N15/81Vectors or expression systems specially adapted for eukaryotic hosts for fungi for yeasts
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/10Cells modified by introduction of foreign genetic material
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/78Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12PFERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P19/00Preparation of compounds containing saccharide radicals
    • C12P19/26Preparation of nitrogen-containing carbohydrates
    • C12P19/28N-glycosides
    • C12P19/30Nucleotides
    • C12P19/34Polynucleotides, e.g. nucleic acids, oligoribonucleotides
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y305/00Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04002Adenine deaminase (3.5.4.2)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y305/00Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04004Adenosine deaminase (3.5.4.4)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3519Fusion with another nucleic acid
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use 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)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Mycology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Cell Biology (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Saccharide Compounds (AREA)
  • User Interface Of Digital Computer (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)

Abstract

RNA GUIA ANTI-SENTIDO COM REGIÃO FUNCIONAL ADICIONADA PARA EDIÇÃO DE RNA ALVO. Objetivo: Fornecer um RNA guia anti-sentido para edição de um RNA alvo por ADAR. Solução: Um RNA guia anti-sentido para edição de um RNA alvo por ADAR, contendo pelo menos uma região funcional e uma região anti-sentido que é complementar a uma porção do RNA alvo e pode formar uma fita dupla com o RNA alvo, na qual a pelo menos uma região funcional é ligada à região anti-sentido, e na qual o RNA guia não contém substancialmente uma sequência de bases de recrutamento de ADAR.
BR112022011277A 2019-12-09 2020-12-08 Rna guia anti-sentido com região funcional adicionada para edição de rna alvo BR112022011277A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
JP2019222437 2019-12-09
PCT/JP2020/045707 WO2021117729A1 (ja) 2019-12-09 2020-12-08 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

Publications (1)

Publication Number Publication Date
BR112022011277A2 true BR112022011277A2 (pt) 2022-09-06

Family

ID=76329853

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022011277A BR112022011277A2 (pt) 2019-12-09 2020-12-08 Rna guia anti-sentido com região funcional adicionada para edição de rna alvo

Country Status (13)

Country Link
US (1) US20230059753A1 (pt)
EP (1) EP4074825A1 (pt)
JP (1) JPWO2021117729A1 (pt)
KR (1) KR20220111322A (pt)
CN (1) CN114787357A (pt)
AU (1) AU2020399673A1 (pt)
BR (1) BR112022011277A2 (pt)
CA (1) CA3161264A1 (pt)
CO (1) CO2022009538A2 (pt)
IL (1) IL293671A (pt)
MX (1) MX2022006991A (pt)
TW (1) TW202136508A (pt)
WO (1) WO2021117729A1 (pt)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115038789A (zh) 2019-12-02 2022-09-09 塑造治疗公司 治疗性编辑
US20240011028A9 (en) * 2020-04-22 2024-01-11 Shape Therapeutics Inc. Compositions and Methods Using SNRNA Components
US20240150754A1 (en) 2020-12-25 2024-05-09 Astellas Pharma Inc. Guide rna for editing polyadenylation signal sequence of target rna
CA3236122A1 (en) * 2021-10-27 2023-05-04 Shape Therapeutics Inc. Engineered rnas
WO2023077128A1 (en) * 2021-10-29 2023-05-04 Board Of Regents, The University Of Texas System Shielded small nucleotides for intracellular barcoding
WO2023152371A1 (en) 2022-02-14 2023-08-17 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
WO2023196853A1 (en) * 2022-04-05 2023-10-12 Astellas Gene Therapies, Inc. Compositions and methods for the treatment of muscular dystrophies
WO2023214512A1 (ja) * 2022-05-06 2023-11-09 学校法人常翔学園 人工rna分子
WO2024013360A1 (en) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Chemically modified oligonucleotides for adar-mediated rna editing
WO2024013361A1 (en) 2022-07-15 2024-01-18 Proqr Therapeutics Ii B.V. Oligonucleotides for adar-mediated rna editing and use thereof
WO2024044282A1 (en) * 2022-08-24 2024-02-29 Shape Therapeutics Inc. Engineered constructs for increased transcription of rna payloads
WO2024078345A1 (zh) * 2022-10-11 2024-04-18 广州瑞风生物科技有限公司 snRNA核酸分子及其应用
GB202215614D0 (en) 2022-10-21 2022-12-07 Proqr Therapeutics Ii Bv Heteroduplex rna editing oligonucleotide complexes

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5814500A (en) * 1996-10-31 1998-09-29 The Johns Hopkins University School Of Medicine Delivery construct for antisense nucleic acids and methods of use
JP4936343B6 (ja) * 2008-10-23 2018-06-27 国立大学法人 東京大学 microRNAの機能阻害法
GB201412802D0 (en) * 2014-07-18 2014-09-03 Univ London Queen Mary Method
WO2016097212A1 (en) 2014-12-17 2016-06-23 Proqr Therapeutics Ii B.V. Targeted rna editing
WO2016143700A1 (ja) 2015-03-06 2016-09-15 タグシクス・バイオ株式会社 Dnaアプタマーの安定化法
JP6624743B2 (ja) 2015-07-14 2019-12-25 学校法人福岡大学 部位特異的rna変異導入方法およびそれに使用する標的編集ガイドrnaならびに標的rna−標的編集ガイドrna複合体
DE102015012522B3 (de) 2015-09-26 2016-06-02 Eberhard Karls Universität Tübingen Verfahren und Substanzen zur gerichteten RNA-Editierung
EP3449002A4 (en) 2016-04-29 2019-09-25 Nanyang Technological University ANTISENSE OLIGONUCLEOTIDES CONTAINING A G-QUADRUPLEX
WO2017220751A1 (en) 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Single-stranded rna-editing oligonucleotides
PL3507366T3 (pl) 2016-09-01 2021-05-04 Proqr Therapeutics Ii B.V. Chemicznie modyfikowane jednoniciowe oligonukleotydy edytujące rna
WO2018134301A1 (en) 2017-01-19 2018-07-26 Proqr Therapeutics Ii B.V. Oligonucleotide complexes for use in rna editing
CA3076740A1 (en) * 2017-10-06 2019-04-11 Oregon Health & Science University Compositions and methods for editing rna
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
JP7333508B2 (ja) * 2017-12-06 2023-08-25 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

Also Published As

Publication number Publication date
EP4074825A1 (en) 2022-10-19
CO2022009538A2 (es) 2022-07-19
JPWO2021117729A1 (pt) 2021-06-17
AU2020399673A1 (en) 2022-07-28
TW202136508A (zh) 2021-10-01
KR20220111322A (ko) 2022-08-09
WO2021117729A1 (ja) 2021-06-17
MX2022006991A (es) 2022-08-25
CA3161264A1 (en) 2021-06-17
US20230059753A1 (en) 2023-02-23
IL293671A (en) 2022-08-01
CN114787357A (zh) 2022-07-22

Similar Documents

Publication Publication Date Title
BR112022011277A2 (pt) Rna guia anti-sentido com região funcional adicionada para edição de rna alvo
BR112021021736A2 (pt) Batente de tecido para um instrumento cirúrgico
BR112019006194A2 (pt) composições que compreendem um ligante de ligação ao receptor de canabinóides
BR112018006920A2 (pt) agonistas dos receptores de glucagon
UY36554A (es) ARNi VARIANTE
BR112019010342A2 (pt) composições do irna de serpina1 e métodos de uso destas
BR112015022776A8 (pt) estruturas de sola e artigos de calçado contendo membros de entressola leves com elementos protetores
CO2021011188A2 (es) Compuesto heterocíclico y su uso
BR112017025749A2 (pt) suporte de seringa e autoinjetor
BR112014030020A2 (pt) aparelho para montagem de seções de pá
CR20170425A (es) Nuevas proteínas específicas para pioverdina y pioquelina
BR112015032484A2 (pt) um artigo para calçados
BR112022021136A2 (pt) Composições de irna de fator b de complemento (cfb) e métodos de uso das mesmas
BR112021020327A2 (pt) Progressão adiabática com re-otimização intermediária para resolver problemas quânticos de variação dura na computação quântica
BR112016028998A2 (pt) composição polimerizável para material óptico e uso da mesma
BR112018016701A2 (pt) uso de uma composição lubrificante
BR112022017822A2 (pt) Composições de irna de cetoexocinase (khk) e métodos de uso das mesmas
BR112022001191A2 (pt) Combinações fungicidas, misturas e composições e usos das mesmas
BR112021026475A2 (pt) Método para transferência por handover condicional, dispositivo terminal e dispositivo de rede
BR112018071618A2 (pt) métodos, composições e usos relacionados aos mesmos
BR112021018793A2 (pt) Oligonucleotídeo modificado, composição farmacêutica compreendendo o mesmo composto para modular a expressão de dux4 e uso
BR112012016617A2 (pt) sistema de elevador com um par de trilhos guia
BR112015018180A2 (pt) composição estereoisomérica de difenoconazol com fitotoxicidade reduzida
AR120341A1 (es) COMPOSICIONES DE AGENTES DE ARNi CONTRA LA HUNTINGTINA (HTT) Y SUS MÉTODOS DE USO
BR112015019795A2 (pt) processo para a preparação de bispiribac de sódio e intermediários do mesmo