PL3234134T3 - Ukierunkowana edycja rna - Google Patents

Ukierunkowana edycja rna

Info

Publication number
PL3234134T3
PL3234134T3 PL15813826T PL15813826T PL3234134T3 PL 3234134 T3 PL3234134 T3 PL 3234134T3 PL 15813826 T PL15813826 T PL 15813826T PL 15813826 T PL15813826 T PL 15813826T PL 3234134 T3 PL3234134 T3 PL 3234134T3
Authority
PL
Poland
Prior art keywords
rna editing
targeted rna
targeted
editing
rna
Prior art date
Application number
PL15813826T
Other languages
English (en)
Inventor
Bart KLEIN
Gerardus Johannes Platenburg
Original Assignee
Proqr Therapeutics Ii B.V.
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=54979679&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=PL3234134(T3) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Priority claimed from GBGB1512467.0A external-priority patent/GB201512467D0/en
Priority claimed from GBGB1512595.8A external-priority patent/GB201512595D0/en
Priority claimed from GBGB1521987.6A external-priority patent/GB201521987D0/en
Application filed by Proqr Therapeutics Ii B.V. filed Critical Proqr Therapeutics Ii B.V.
Publication of PL3234134T3 publication Critical patent/PL3234134T3/pl

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    • 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
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    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6806Preparing nucleic acids for analysis, e.g. for polymerase chain reaction [PCR] assay
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/16Aptamers
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3519Fusion with another nucleic acid
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/533Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Analytical Chemistry (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Saccharide Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
PL15813826T 2014-12-17 2015-12-17 Ukierunkowana edycja rna PL3234134T3 (pl)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
GB201422511 2014-12-17
GBGB1512467.0A GB201512467D0 (en) 2015-07-16 2015-07-16 Editing
GBGB1512595.8A GB201512595D0 (en) 2015-07-17 2015-07-17 Editing
GBGB1521987.6A GB201521987D0 (en) 2015-12-14 2015-12-14 Editing
EP15813826.3A EP3234134B1 (en) 2014-12-17 2015-12-17 Targeted rna editing
PCT/EP2015/080347 WO2016097212A1 (en) 2014-12-17 2015-12-17 Targeted rna editing

Publications (1)

Publication Number Publication Date
PL3234134T3 true PL3234134T3 (pl) 2020-12-28

Family

ID=54979679

Family Applications (1)

Application Number Title Priority Date Filing Date
PL15813826T PL3234134T3 (pl) 2014-12-17 2015-12-17 Ukierunkowana edycja rna

Country Status (13)

Country Link
US (3) US10676737B2 (pl)
EP (2) EP3712269A1 (pl)
JP (1) JP6718872B2 (pl)
CN (1) CN107109413B (pl)
AU (2) AU2015367378A1 (pl)
BR (1) BR112017011510B1 (pl)
CA (1) CA2968336C (pl)
DK (1) DK3234134T3 (pl)
ES (1) ES2810375T3 (pl)
IL (1) IL252386B (pl)
PL (1) PL3234134T3 (pl)
RU (1) RU2711506C2 (pl)
WO (1) WO2016097212A1 (pl)

Families Citing this family (151)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013066438A2 (en) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
ES2810375T3 (es) 2014-12-17 2021-03-08 Proqr Therapeutics Ii Bv Edición direccionada de ARN
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
SG10202104041PA (en) 2015-10-23 2021-06-29 Harvard College Nucleobase editors and uses thereof
US10370667B2 (en) 2015-11-18 2019-08-06 Rosalind Franklin University Of Medicine And Science Antisense compounds targeting leucine-rich repeat kinase 2 (LRRK2) for the treatment of parkinsons disease
US9840710B2 (en) 2015-11-18 2017-12-12 Rosalind Franklin University Of Medicine And Science Antisense compounds targeting leucine-rich repeat kinase 2 (LRRK2) for the treatment of parkinsons disease
WO2017091630A1 (en) 2015-11-23 2017-06-01 The Regents Of The University Of California Tracking and manipulating cellular rna via nuclear delivery of crispr/cas9
AU2017205462A1 (en) 2016-01-05 2018-06-07 Ionis Pharmaceuticals, Inc. Methods for reducing LRRK2 expression
EP3475424A1 (en) 2016-06-22 2019-05-01 ProQR Therapeutics II B.V. Single-stranded rna-editing oligonucleotides
KR20250103795A (ko) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
CA3035293A1 (en) * 2016-09-01 2018-03-08 Proqr Therapeutics Ii B.V. Chemically modified single-stranded rna-editing oligonucleotides
SG11201903089RA (en) 2016-10-14 2019-05-30 Harvard College Aav delivery of nucleobase editors
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
EP3571300A1 (en) 2017-01-19 2019-11-27 ProQR Therapeutics II B.V. Oligonucleotide complexes for use in rna editing
DK3582609T3 (da) * 2017-02-15 2026-02-09 Keygene Nv Fremgangsmåder til målrettet genetisk ændring i planteceller
CN118416088A (zh) 2017-03-03 2024-08-02 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
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
EP3592381A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Cancer vaccine
WO2018165629A1 (en) 2017-03-10 2018-09-13 President And Fellows Of Harvard College Cytosine to guanine base editor
WO2018176009A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
CA3062595A1 (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
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
CN111727247A (zh) 2017-10-04 2020-09-29 博德研究所 用于靶向核酸编辑的系统、方法和组合物
US11795443B2 (en) 2017-10-16 2023-10-24 The Broad Institute, Inc. Uses of adenosine base editors
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
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
CN108165543B (zh) * 2017-12-26 2021-01-22 山东省农业科学院生物技术研究中心 水稻腺苷脱氨酶OsAD1及其编码基因在叶绿体基因RNA编辑中的应用
WO2019158475A1 (en) 2018-02-14 2019-08-22 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for rna editing
EP3790964A4 (en) * 2018-05-11 2022-06-08 Beam Therapeutics, Inc. METHODS FOR REMOVAL OF PATHOGENIC MUTATIONS USING PROGRAMMABLE BASE EDITOR SYSTEMS
AU2019265019B2 (en) 2018-05-11 2025-11-06 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
CN108595914B (zh) * 2018-05-16 2021-06-25 湖南农业大学 一种烟草线粒体rna编辑位点高精度预测方法
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
CN110527697B (zh) * 2018-05-23 2023-07-07 中国科学院分子植物科学卓越创新中心 基于CRISPR-Cas13a的RNA定点编辑技术
US11541105B2 (en) 2018-06-01 2023-01-03 The Research Foundation For The State University Of New York Compositions and methods for disrupting biofilm formation and maintenance
TWI833770B (zh) 2018-06-27 2024-03-01 美商Ionis製藥公司 用於減少 lrrk2 表現之化合物及方法
JP7347830B2 (ja) * 2018-06-29 2023-09-20 エバーハルト カール ウニヴェルジテート テュービンゲン Rna編集のための人工核酸
EP3820495A4 (en) 2018-07-09 2022-07-20 The Broad Institute Inc. RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES
EP3844274A1 (en) 2018-08-28 2021-07-07 Roche Innovation Center Copenhagen A/S Neoantigen engineering using splice modulating compounds
US20220010333A1 (en) * 2018-09-06 2022-01-13 The Regents Of The University Of California Rna and dna base editing via engineered adar recruitment
EP3850088A4 (en) 2018-09-07 2023-07-19 Beam Therapeutics, Inc. Compositions and methods for improving base editing
AU2019336245B2 (en) 2018-09-07 2026-02-26 Beam Therapeutics Inc. Compositions and methods for delivering a nucleobase editing system
EP4527931A1 (en) * 2018-10-12 2025-03-26 Peking University Methods and compositions for editing rnas
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
CA3126947A1 (en) 2019-01-22 2020-07-30 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
JP2022519184A (ja) 2019-01-22 2022-03-22 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
CA3127243A1 (en) 2019-01-22 2020-07-30 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020157008A1 (en) 2019-01-28 2020-08-06 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
AU2020223314A1 (en) 2019-02-13 2021-08-12 Beam Therapeutics Inc. Compositions and methods for treating alpha-1 antitrypsin deficiency
SG11202108346WA (en) 2019-02-13 2021-08-30 Beam Therapeutics Inc Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence
EP3942043A2 (en) 2019-03-19 2022-01-26 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
GB201904709D0 (en) 2019-04-03 2019-05-15 Proqr Therapeutics Ii Bv Chemically modified oligonucleotides
PH12021552613A1 (en) 2019-04-15 2022-06-27 Edigene Therapeutics Beijing Inc METHODS AND COMPOSITIONS FOR EDITING RNAs
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
GB201905732D0 (en) 2019-04-24 2019-06-05 Proqr Therapeutics Ii Bv RNA editing inhibitors and methods of use
US11680109B2 (en) 2019-05-29 2023-06-20 Duke University Compositions and methods for detection and measurement of RNA modifications through targeted RNA editing
AU2020287787A1 (en) * 2019-06-05 2022-01-20 Daiichi Sankyo Company, Limited Stable target-editing guide RNA having chemically modified nucleic acid introduced thereinto
AU2020290517B2 (en) 2019-06-13 2026-02-26 Proqr Therapeutics Ii B.V. Antisense RNA editing oligonucleotides comprising cytidine analogs
CN114269919B (zh) 2019-07-12 2023-08-18 北京大学 采用工程化rna利用内源adar进行靶向的rna编辑
US20220275359A1 (en) 2019-08-01 2022-09-01 Astellas Pharma Inc. Guide rna for editing target, with functional nucleotide sequence added
CN115279398B (zh) 2019-09-27 2025-07-04 比姆医疗股份有限公司 治疗液态癌症的组合物和方法
CN114514318B (zh) * 2019-09-27 2025-01-10 弗雷斯特公司 寡核苷酸和靶rna的位点特异性编辑方法
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
AU2020395113A1 (en) 2019-12-02 2022-06-09 Shape Therapeutics Inc. Therapeutic editing
JP7754721B2 (ja) 2019-12-09 2025-10-15 アステラス製薬株式会社 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
JP7765837B2 (ja) 2019-12-23 2025-11-07 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 シュタルガルト病の処置におけるヌクレオチド脱アミノ化のためのアンチセンスオリゴヌクレオチド
PH12022551595A1 (en) * 2019-12-30 2023-11-29 Edigene Therapeutics Beijing Inc Leaper technology based method for treating mps ih and composition
CN113122524B (zh) * 2019-12-31 2023-03-24 北京辑因医疗科技有限公司 一种靶向编辑rna的新方法
US20240247258A1 (en) * 2020-03-12 2024-07-25 Frest Inc. Oligonucleotide and target rna site-specific editing method
WO2021216853A1 (en) * 2020-04-22 2021-10-28 Shape Therapeutics Inc. Compositions and methods using snrna components
BR112022022603A2 (pt) 2020-05-08 2023-01-17 Broad Inst Inc Métodos e composições para edição simultânea de ambas as fitas de sequência alvo de nucleotídeos de fita dupla
WO2021231679A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of gap junction protein beta 2 (gjb2)
EP4150077A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of transmembrane channel-like protein 1 (tmc1)
WO2021231698A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl)
WO2021231691A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of retinoschisin 1 (rsi)
EP4150088A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate synthetase (ass1)
WO2021231692A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of otoferlin (otof)
WO2021231680A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of methyl-cpg binding protein 2 (mecp2)
WO2021231673A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of leucine rich repeat kinase 2 (lrrk2)
WO2021242870A1 (en) * 2020-05-26 2021-12-02 Shape Therapeutics Inc. Compositions and methods for genome editing
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
AU2021280283A1 (en) * 2020-05-26 2022-12-08 Shape Therapeutics Inc. Compositions and methods for modifying target RNAs
JP2023532375A (ja) * 2020-07-06 2023-07-27 北京▲輯▼因医▲療▼科技有限公司 改良されたrna編集方法
GB202011428D0 (en) 2020-07-23 2020-09-09 Proqr Therapeutics Ii Bv Antisense oligonucleotides for RNA editing
CA3190477A1 (en) * 2020-07-30 2022-02-03 Adarx Pharmaceuticals, Inc. Adar dependent editing compositions and methods of use thereof
US12576151B2 (en) 2020-09-25 2026-03-17 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
WO2022078569A1 (en) 2020-10-12 2022-04-21 Eberhard Karls Universität Tübingen Artificial nucleic acids for rna editing
CN114380918B (zh) * 2020-10-19 2023-03-31 上海交通大学 一种目标rna单碱基编辑的系统和方法
IL303533A (en) 2020-12-08 2023-08-01 Univ Fukuoka RNA is a stable guide for editing targets into which chemically modified nucleic acids are inserted
US20240150754A1 (en) * 2020-12-25 2024-05-09 Astellas Pharma Inc. Guide rna for editing polyadenylation signal sequence of target rna
WO2022150974A1 (en) * 2021-01-12 2022-07-21 Peking University Targeted rna editing by leveraging endogenous adar using engineered rnas
WO2022221457A1 (en) * 2021-04-13 2022-10-20 Adarx Pharmaceuticals, Inc. Rna silencing agents and methods of use
GB202107553D0 (en) 2021-05-27 2021-07-14 Univ Oxford Innovation Ltd Method
WO2022256283A2 (en) 2021-06-01 2022-12-08 Korro Bio, Inc. Methods for restoring protein function using adar
EP4405478A1 (en) 2021-09-20 2024-07-31 Alnylam Pharmaceuticals, Inc. Inhibin subunit beta e (inhbe) modulator compositions and methods of use thereof
EP4408999A1 (en) 2021-09-29 2024-08-07 F. Hoffmann-La Roche AG Rna editing
AU2022370009A1 (en) 2021-10-22 2024-05-16 Korro Bio, Inc. Methods and compositions for disrupting nrf2-keap1 protein interaction by adar mediated rna editing
US20250084408A1 (en) * 2021-10-27 2025-03-13 Shape Therapeutics Inc. Engineered rnas
WO2023102449A2 (en) * 2021-12-01 2023-06-08 Shape Therapeutics Inc. Engineered guide rnas and polynucleotides
US20250154504A1 (en) 2022-02-14 2025-05-15 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
EP4555086A1 (en) 2022-07-15 2025-05-21 ProQR Therapeutics II B.V. Oligonucleotides for adar-mediated rna editing and use thereof
EP4555085A1 (en) 2022-07-15 2025-05-21 ProQR Therapeutics II B.V. Chemically modified oligonucleotides for adar-mediated 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
US20250101418A1 (en) 2022-11-30 2025-03-27 Eberhard-Karls-Universität-Tübingen Chemically modified antisense oligonucleotides (asos) and compositions comprising the same for rna editing
GB202218090D0 (en) 2022-12-01 2023-01-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency
CN120322556A (zh) 2022-12-09 2025-07-15 ProQR治疗上市公司Ⅱ 用于治疗心血管疾病的反义寡核苷酸
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
EP4432289A1 (en) 2023-03-15 2024-09-18 Shape Therapeutics Inc. Generative sequence screening with conditional gans, diffusion models, and denoising diffusion conditional gans
WO2024196779A2 (en) * 2023-03-17 2024-09-26 The Board Of Trustees Of The Leland Stanford Junior University Optimized antisense sequences that enhance targeted rna editing by adar proteins
JP2026510408A (ja) 2023-03-24 2026-04-02 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 神経障害の処置のためのアンチセンスオリゴヌクレオチド
GB202304363D0 (en) 2023-03-24 2023-05-10 Proqr Therapeutics Ii Bv Chemically modified antisense oligonucleotides for use in RNA editing
CN121002181A (zh) 2023-03-27 2025-11-21 ProQR治疗上市公司Ⅱ 用于治疗肝脏疾病的反义寡核苷酸
EP4698645A1 (en) 2023-04-21 2026-02-25 University of Rochester Nucleic acid molecules for pseudouridylation
AR132964A1 (es) 2023-06-16 2025-08-13 Proqr Therapeutics Ii Bv Oligonucleótidos antisentido para el tratamiento de enfermedades neurodegenerativas
WO2025047679A1 (ja) 2023-08-25 2025-03-06 学校法人福岡大学 リン酸部修飾を導入した安定型標的編集ガイドrna
WO2025051946A1 (en) 2023-09-07 2025-03-13 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of metabolic disorders
WO2025073902A1 (en) 2023-10-06 2025-04-10 Eberhard Karls Universität Tübingen Chemical and sequence optimisation of antisense oligonucleotides for adar-mediated rna editing
WO2025083268A1 (en) 2023-10-20 2025-04-24 Airna Corporation Chemically modified antisense oligonucleotides (asos) and compositions comprising the same for rna editing
AU2024379264A1 (en) 2023-11-16 2026-05-14 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
WO2025155911A1 (en) 2024-01-18 2025-07-24 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of hurler syndrome
GB202404661D0 (en) 2024-04-02 2024-05-15 Proqr Therapeutics Ii Bv Antisense oligoncleotides for the treatment of liver disease
GB202405143D0 (en) 2024-04-11 2024-05-29 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of poly-q disease
WO2025224230A1 (en) 2024-04-25 2025-10-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of fatty liver disease
WO2025231085A1 (en) 2024-04-30 2025-11-06 Airna Corporation Site-directed editing of rna
WO2026006436A1 (en) 2024-06-25 2026-01-02 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of tar dna binding protein 43 kda (tdp43)
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
WO2026077951A1 (en) 2024-10-07 2026-04-16 Vico Therapeutics B.V. Improved aon for rna editing
WO2026080897A1 (en) 2024-10-11 2026-04-16 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of chronic pain
WO2026093339A1 (en) 2024-10-29 2026-05-07 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of peroxisome biogenesis disorders in the zellweger spectrum (pbd-zsd)

Family Cites Families (49)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE2330003A1 (de) 1973-06-13 1975-01-09 Rudolf Neumann Verstellbarer netzhalter mit druckrollen oder kloetzen
NZ514348A (en) 1999-05-04 2004-05-28 Exiqon As L-ribo-LNA analogues
CA2521464C (en) 2003-04-09 2013-02-05 Alnylam Pharmaceuticals, Inc. Irna conjugates
US20050074801A1 (en) 2003-09-09 2005-04-07 Monia Brett P. Chimeric oligomeric compounds comprising alternating regions of northern and southern conformational geometry
WO2005087949A1 (en) 2004-03-12 2005-09-22 Compugen Ltd. Systematic mapping of adenosine to inosine editing sites in the human transcriptome
WO2005094370A2 (en) 2004-03-29 2005-10-13 The General Hospital Corporation Oligonucleotide complex compositions and methods of use as gene alteration tools
WO2005111211A2 (en) * 2004-05-14 2005-11-24 Rosetta Genomics Ltd. Micronas and uses thereof
US7834064B2 (en) * 2005-01-03 2010-11-16 Andres Mario Salazar Clinical method for the immunomodulatory and vaccine adjuvant use of poly-ICLC and other dsRNAs
JP5252622B2 (ja) 2007-01-17 2013-07-31 独立行政法人産業技術総合研究所 高いヌクレアーゼ耐性と優れたrna干渉効果を発現可能な二本鎖rna
SG171914A1 (en) 2008-12-02 2011-07-28 Chiralgen Ltd Method for the synthesis of phosphorus atom modified nucleic acids
ES2628144T3 (es) 2009-04-03 2017-08-01 Dicerna Pharmaceuticals, Inc. Métodos y composiciones para la inhibición específica de KRAS por RNA bicatenario asimétrico
EP2451461A4 (en) 2009-07-06 2013-05-29 Ontorii Inc NOVEL NUCLEIC ACID PRODRUGS AND METHOD OF USE THEREOF
JP5685267B2 (ja) 2009-12-09 2015-03-18 日東電工株式会社 Hsp47発現の調節
BR112012024049A2 (pt) 2010-03-24 2017-03-01 Rxi Pharmaceuticals Corp interferência de rna em indicações dérmicas e fibróticas
CA2804210A1 (en) 2010-07-06 2012-01-12 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of androgen receptor by double-stranded rna
US20140128449A1 (en) 2011-04-07 2014-05-08 The Board Of Regents Of The University Of Texas System Oligonucleotide modulation of splicing
US9238814B2 (en) 2011-05-20 2016-01-19 Fukuoka University Ribozyme for identifying modification on RNA sequence and RNA cleavage method using same
MY167390A (en) 2011-11-18 2018-08-16 Alnylam Pharmaceuticals Inc Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases
US9637739B2 (en) * 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
US9605255B2 (en) 2012-07-12 2017-03-28 Proqr Therapeutics Ii B.V. Oligonucleotides for making a change in the sequence of a target RNA molecule present in a living cell
CN112007045B (zh) 2012-07-13 2026-01-06 波涛生命科学有限公司 手性控制
DK2872485T3 (da) 2012-07-13 2021-03-08 Wave Life Sciences Ltd Asymmetrisk hjælpegruppe
US9650627B1 (en) 2012-07-19 2017-05-16 University Of Puerto Rico Site-directed RNA editing
KR102463973B1 (ko) * 2013-05-22 2022-11-07 알닐람 파마슈티칼스 인코포레이티드 SERPINA1 iRNA 조성물 및 이의 사용 방법
EP3010514B1 (en) 2013-06-16 2021-01-20 National University Corporation Tokyo Medical and Dental University Double-stranded antisense nucleic acid with exon-skipping effect
KR102423317B1 (ko) 2014-01-16 2022-07-22 웨이브 라이프 사이언시스 리미티드 키랄 디자인
GB201418892D0 (en) 2014-10-23 2014-12-10 Proqr Therapeutics B V DNA editing
JP2017536119A (ja) 2014-11-19 2017-12-07 ロシュ イノベーション センター コペンハーゲン エーエス キラルホスホロチオエート連結を含むlnaギャップマーオリゴヌクレオチド
WO2016089433A1 (en) 2014-12-03 2016-06-09 Agilent Technologies, Inc. Guide rna with chemical modifications
US12359197B2 (en) 2014-12-12 2025-07-15 Etagen Pharma, Inc. Compositions and methods for editing nucleic acids in cells utilizing oligonucleotides
US10815481B2 (en) 2014-12-16 2020-10-27 Roche Innovation Center Copenhagen A/S Chiral library screen
ES2810375T3 (es) 2014-12-17 2021-03-08 Proqr Therapeutics Ii Bv Edición direccionada de ARN
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
WO2017015575A1 (en) 2015-07-22 2017-01-26 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
DE102015012522B3 (de) 2015-09-26 2016-06-02 Eberhard Karls Universität Tübingen Verfahren und Substanzen zur gerichteten RNA-Editierung
EP3359523A4 (en) 2015-10-09 2019-07-24 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND METHOD THEREFOR
US20190249173A1 (en) 2016-05-04 2019-08-15 Chandra Vargeese Methods and compositions of biologically active agents
JP2019516680A (ja) 2016-05-04 2019-06-20 ウェイブ ライフ サイエンシズ リミテッドWave Life Sciences Ltd. オリゴヌクレオチド組成物およびその方法
EP3458464B1 (en) 2016-05-18 2026-04-15 ETH Zurich Stereoselective synthesis of phosphorothioate oligoribonucleotides
US10767175B2 (en) 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
EP3475424A1 (en) 2016-06-22 2019-05-01 ProQR Therapeutics II B.V. Single-stranded rna-editing oligonucleotides
CA3035293A1 (en) 2016-09-01 2018-03-08 Proqr Therapeutics Ii B.V. Chemically modified single-stranded rna-editing oligonucleotides
EP3571300A1 (en) * 2017-01-19 2019-11-27 ProQR Therapeutics II B.V. Oligonucleotide complexes for use in rna editing
CN118416088A (zh) * 2017-03-03 2024-08-02 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
CA3064601A1 (en) 2017-06-26 2019-01-03 President And Fellows Of Harvard College Crispr/cas-adenine deaminase based compositions, systems, and methods for targeted nucleic acid editing
WO2019158475A1 (en) 2018-02-14 2019-08-22 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for rna editing
CA3095311A1 (en) 2018-03-27 2019-10-03 University Of Rochester Nucleic acid molecules for pseudouridylation
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
AU2020290517B2 (en) 2019-06-13 2026-02-26 Proqr Therapeutics Ii B.V. Antisense RNA editing oligonucleotides comprising cytidine analogs

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