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 alvoInfo
- 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
Links
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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
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing nucleic acids
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/50—Hydrolases (3) acting on carbon-nitrogen bonds, other than peptide bonds (3.5), e.g. asparaginase
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- 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
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- 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
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- C12N15/09—Recombinant DNA-technology
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- C12N15/09—Recombinant DNA-technology
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- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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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
- 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
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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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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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
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- C12N15/80—Vectors or expression systems specially adapted for eukaryotic hosts for fungi
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- 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
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
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- C12P19/00—Preparation of compounds containing saccharide radicals
- C12P19/26—Preparation of nitrogen-containing carbohydrates
- C12P19/28—N-glycosides
- C12P19/30—Nucleotides
- C12P19/34—Polynucleotides, e.g. nucleic acids, oligoribonucleotides
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- C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
- C12Y305/04002—Adenine deaminase (3.5.4.2)
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- C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
- C12Y305/04004—Adenosine deaminase (3.5.4.4)
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- A—HUMAN NECESSITIES
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
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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
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- 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
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
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- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
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- Bioinformatics & Cheminformatics (AREA)
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- Wood Science & Technology (AREA)
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- General Engineering & Computer Science (AREA)
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- Biochemistry (AREA)
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- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Medicinal Chemistry (AREA)
- Epidemiology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
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- 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)
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- 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.
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)
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)
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 |
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2020
- 2020-12-08 BR BR112022011277A patent/BR112022011277A2/pt unknown
- 2020-12-08 AU AU2020399673A patent/AU2020399673A1/en active Pending
- 2020-12-08 TW TW109143300A patent/TW202136508A/zh unknown
- 2020-12-08 EP EP20899155.4A patent/EP4074825A1/en active Pending
- 2020-12-08 JP JP2021563975A patent/JPWO2021117729A1/ja active Pending
- 2020-12-08 CA CA3161264A patent/CA3161264A1/en active Pending
- 2020-12-08 KR KR1020227023235A patent/KR20220111322A/ko unknown
- 2020-12-08 WO PCT/JP2020/045707 patent/WO2021117729A1/ja unknown
- 2020-12-08 IL IL293671A patent/IL293671A/en unknown
- 2020-12-08 CN CN202080084942.6A patent/CN114787357A/zh active Pending
- 2020-12-08 MX MX2022006991A patent/MX2022006991A/es unknown
- 2020-12-08 US US17/783,591 patent/US20230059753A1/en active Pending
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2022
- 2022-07-07 CO CONC2022/0009538A patent/CO2022009538A2/es unknown
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 |
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