PL3507366T3 - Chemicznie modyfikowane jednoniciowe oligonukleotydy edytujące rna - Google Patents
Chemicznie modyfikowane jednoniciowe oligonukleotydy edytujące rnaInfo
- Publication number
- PL3507366T3 PL3507366T3 PL17771348T PL17771348T PL3507366T3 PL 3507366 T3 PL3507366 T3 PL 3507366T3 PL 17771348 T PL17771348 T PL 17771348T PL 17771348 T PL17771348 T PL 17771348T PL 3507366 T3 PL3507366 T3 PL 3507366T3
- Authority
- PL
- Poland
- Prior art keywords
- uniconious
- chemically modified
- rna editing
- editing oligonucleotides
- oligonucleotides
- Prior art date
Links
- 108091034117 Oligonucleotide Proteins 0.000 title 1
- JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Polymers Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 title 1
Classifications
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- C—CHEMISTRY; METALLURGY
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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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- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
- A61P11/06—Antiasthmatics
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- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
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- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
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- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
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- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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- A61P37/02—Immunomodulators
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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- A—HUMAN NECESSITIES
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- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/06—Antianaemics
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- 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)
- C12Y305/04004—Adenosine deaminase (3.5.4.4)
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- 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
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- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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- 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
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3521—Methyl
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- 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
- C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
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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
- C12N2320/34—Allele or polymorphism specific uses
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- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Veterinary Medicine (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- General Chemical & Material Sciences (AREA)
- Public Health (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Biomedical Technology (AREA)
- Genetics & Genomics (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Immunology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Physical Education & Sports Medicine (AREA)
- Pulmonology (AREA)
- Psychology (AREA)
- Dermatology (AREA)
- Pain & Pain Management (AREA)
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Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GBGB1614858.7A GB201614858D0 (en) | 2016-09-01 | 2016-09-01 | Modified Bulgeomers |
| GBGB1616374.3A GB201616374D0 (en) | 2016-09-27 | 2016-09-27 | Modified bulgeomers |
| GBGB1621467.8A GB201621467D0 (en) | 2016-12-16 | 2016-12-16 | Modified bulgeomers |
| GBGB1703034.7A GB201703034D0 (en) | 2017-02-24 | 2017-02-24 | Modified bulgeomers |
| GBGB1707508.6A GB201707508D0 (en) | 2017-05-10 | 2017-05-10 | Modified bulgeomers |
| EP17771348.4A EP3507366B1 (en) | 2016-09-01 | 2017-08-31 | Chemically modified single-stranded rna-editing oligonucleotides |
| PCT/EP2017/071912 WO2018041973A1 (en) | 2016-09-01 | 2017-08-31 | Chemically modified single-stranded rna-editing oligonucleotides |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PL3507366T3 true PL3507366T3 (pl) | 2021-05-04 |
Family
ID=59923385
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PL17771348T PL3507366T3 (pl) | 2016-09-01 | 2017-08-31 | Chemicznie modyfikowane jednoniciowe oligonukleotydy edytujące rna |
Country Status (15)
| Country | Link |
|---|---|
| US (4) | US10941402B2 (pl) |
| EP (1) | EP3507366B1 (pl) |
| JP (1) | JP7244922B2 (pl) |
| KR (1) | KR102501980B1 (pl) |
| CN (1) | CN110352244B (pl) |
| AU (1) | AU2017320901C1 (pl) |
| CA (1) | CA3035293A1 (pl) |
| DK (1) | DK3507366T3 (pl) |
| ES (1) | ES2837076T3 (pl) |
| IL (1) | IL264923B2 (pl) |
| NZ (1) | NZ751483A (pl) |
| PL (1) | PL3507366T3 (pl) |
| PT (1) | PT3507366T (pl) |
| WO (1) | WO2018041973A1 (pl) |
| ZA (1) | ZA201901016B (pl) |
Families Citing this family (111)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12359197B2 (en) * | 2014-12-12 | 2025-07-15 | Etagen Pharma, Inc. | Compositions and methods for editing nucleic acids in cells utilizing oligonucleotides |
| WO2016097212A1 (en) * | 2014-12-17 | 2016-06-23 | Proqr Therapeutics Ii B.V. | Targeted rna editing |
| EP3929293A3 (en) | 2015-04-03 | 2022-03-16 | University Of Massachusetts | Fully stabilized asymmetric sirna |
| US10633653B2 (en) | 2015-08-14 | 2020-04-28 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| US10478503B2 (en) | 2016-01-31 | 2019-11-19 | University Of Massachusetts | Branched oligonucleotides |
| WO2017220751A1 (en) | 2016-06-22 | 2017-12-28 | Proqr Therapeutics Ii B.V. | Single-stranded rna-editing oligonucleotides |
| KR102501980B1 (ko) | 2016-09-01 | 2023-02-20 | 프로큐알 테라퓨틱스 Ⅱ 비.브이. | 화학적으로 변형된 단일 가닥 rna-편집 올리고뉴클레오타이드 |
| EP3571300A1 (en) | 2017-01-19 | 2019-11-27 | ProQR Therapeutics II B.V. | Oligonucleotide complexes for use in rna editing |
| US11932856B2 (en) * | 2017-03-03 | 2024-03-19 | The Regents Of The University Of California | RNA targeting of mutations via suppressor tRNAs and deaminases |
| US10844377B2 (en) | 2017-06-23 | 2020-11-24 | University Of Massachusetts | Two-tailed self-delivering siRNA |
| CN118421665A (zh) * | 2017-10-06 | 2024-08-02 | 俄勒冈健康与科学大学 | 用于编辑rna的组合物和方法 |
| EP3752611A1 (en) | 2018-02-14 | 2020-12-23 | ProQR Therapeutics II B.V. | Antisense oligonucleotides for rna editing |
| JP7642531B2 (ja) | 2018-05-11 | 2025-03-10 | ビーム セラピューティクス インク． | プログラム可能塩基エディターシステムを用いて病原性アミノ酸を置換する方法 |
| US20210371858A1 (en) * | 2018-05-11 | 2021-12-02 | Beam Therapeutics Inc. | Methods of suppressing pathogenic mutations using programmable base editor systems |
| WO2019217943A1 (en) * | 2018-05-11 | 2019-11-14 | Beam Therapeutics Inc. | Methods of editing single nucleotide polymorphism using programmable base editor systems |
| GB201808146D0 (en) * | 2018-05-18 | 2018-07-11 | Proqr Therapeutics Ii Bv | Stereospecific Linkages in RNA Editing Oligonucleotides |
| CN112752844A (zh) * | 2018-06-29 | 2021-05-04 | 蒂宾根大学 | 用于rna编辑的人工核酸 |
| KR102916447B1 (ko) | 2018-08-10 | 2026-01-23 | 유니버시티 오브 매사추세츠 | Snp를 표적화하는 변형된 올리고뉴클레오티드 |
| JP7627042B2 (ja) | 2018-08-23 | 2025-02-05 | ユニバーシティー オブ マサチューセッツ | Ｏ－メチルリッチ完全安定化オリゴヌクレオチド |
| US20220160870A1 (en) | 2018-08-28 | 2022-05-26 | Roche Innovation Center Copenhagen A/S | Neoantigen engineering using splice modulating compounds |
| EP3847254A4 (en) | 2018-09-07 | 2022-08-10 | Beam Therapeutics Inc. | COMPOSITIONS AND METHODS FOR DELIVERING A NUCLEOBASE EDITING SYSTEM |
| WO2020051562A2 (en) | 2018-09-07 | 2020-03-12 | Beam Therapeutics Inc. | Compositions and methods for improving base editing |
| BR112021006844A8 (pt) * | 2018-10-12 | 2023-03-21 | Univ Beijing | Métodos para editar um rna alvo e para tratar ou prevenir uma doença ou condição, rna editado ou uma célula hospedeira tendo um rna editado, rna que recruta desaminase, construção, biblioteca, composição, célula hospedeira, e, kit para editar um rna alvo . |
| WO2020150636A1 (en) | 2019-01-18 | 2020-07-23 | University Of Massachusetts | Dynamic pharmacokinetic-modifying anchors |
| AU2020211949B2 (en) * | 2019-01-22 | 2025-04-24 | Korro Bio, Inc. | RNA-editing oligonucleotides and uses thereof |
| JP7629404B2 (ja) * | 2019-01-22 | 2025-02-13 | コロ バイオ， インコーポレイテッド | Ｒｎａ編集オリゴヌクレオチド及びその使用 |
| KR20210129646A (ko) * | 2019-01-22 | 2021-10-28 | 코로 바이오, 인크. | Rna-편집 올리고뉴클레오타이드 및 그의 용도 |
| AU2020215232A1 (en) | 2019-01-28 | 2021-08-26 | Proqr Therapeutics Ii B.V. | RNA-editing oligonucleotides for the treatment of usher syndrome |
| GB201901873D0 (en) | 2019-02-11 | 2019-04-03 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for nucleic acid editing |
| WO2020198509A2 (en) | 2019-03-26 | 2020-10-01 | University Of Massachusetts | Modified oligonucleotides with increased stability |
| GB201904709D0 (en) | 2019-04-03 | 2019-05-15 | Proqr Therapeutics Ii Bv | Chemically modified oligonucleotides |
| AU2020259548B2 (en) * | 2019-04-15 | 2023-10-12 | Edigene Therapeutics (Beijing) Inc. | Methods and compositions for editing RNAs |
| GB201905732D0 (en) | 2019-04-24 | 2019-06-05 | Proqr Therapeutics Ii Bv | RNA editing inhibitors and methods of use |
| KR20220016876A (ko) * | 2019-06-05 | 2022-02-10 | 각꼬우호우진 후쿠오카다이가쿠 | 화학 수식 핵산을 도입한 안정형 표적 편집 가이드 rna |
| US20220307023A1 (en) * | 2019-06-13 | 2022-09-29 | Proqr Therapeutics Ii B.V. | Antisense rna editing oligonucleotides comprising cytidine analogs |
| AU2020313143B2 (en) | 2019-07-12 | 2024-04-04 | Peking University | Targeted RNA editing by leveraging endogenous adar using engineered RNAs |
| CN114174510A (zh) | 2019-08-01 | 2022-03-11 | 安斯泰来制药株式会社 | 添加了功能性碱基序列的靶标编辑指导rna |
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