ECSP21080618A - Methods and Compositions for Editing RNAs - Google Patents

Methods and Compositions for Editing RNAs

Info

Publication number
ECSP21080618A
ECSP21080618A ECSENADI202180618A ECDI202180618A ECSP21080618A EC SP21080618 A ECSP21080618 A EC SP21080618A EC SENADI202180618 A ECSENADI202180618 A EC SENADI202180618A EC DI202180618 A ECDI202180618 A EC DI202180618A EC SP21080618 A ECSP21080618 A EC SP21080618A
Authority
EC
Ecuador
Prior art keywords
editing
methods
compositions
rna
anns
Prior art date
Application number
ECSENADI202180618A
Other languages
English (en)
Inventor
Wensheng Wei
Pengfei Yuan
Chunhui Wang
Gangbin Tang
Zhongzheng Cao
Zongyi Yi
Shiyou Zhu
Zhuo Zhou
Nengyin Liu
Zexuan Yi
Yanxia Zhao
Liang Qu
Original Assignee
Univ Beijing
Edigene 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 Univ Beijing, Edigene Inc filed Critical Univ Beijing
Publication of ECSP21080618A publication Critical patent/ECSP21080618A/es

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
    • C12N15/11DNA 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
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing 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
    • C12N15/87Introduction 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
    • 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)
    • 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
    • C12N2310/16Aptamers
    • 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 [CRISPR]
    • 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
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • 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
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521Methyl
    • 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
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin
    • 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
    • C12N2310/50Physical 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

Landscapes

  • Genetics & Genomics (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Compositions Of Macromolecular Compounds (AREA)

Abstract

La presente solicitud proporciona métodos para editar ARN mediante la introducción de un ARN de reclutamiento de desaminasas en una célula hospedera para la desaminación de una adenosina en un ARN objetivo; la presente solicitud proporciona además ARN de reclutamiento de desaminasas usados en los métodos de edición de ARN y composiciones que los comprenden.
ECSENADI202180618A 2019-04-15 2021-11-15 Methods and Compositions for Editing RNAs ECSP21080618A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
CN2019082713 2019-04-15
CN2019129952 2019-12-30
PCT/CN2020/084922 WO2020211780A1 (en) 2019-04-15 2020-04-15 Methods and compositions for editing rnas

Publications (1)

Publication Number Publication Date
ECSP21080618A true ECSP21080618A (es) 2021-12-30

Family

ID=72837012

Family Applications (1)

Application Number Title Priority Date Filing Date
ECSENADI202180618A ECSP21080618A (es) 2019-04-15 2021-11-15 Methods and Compositions for Editing RNAs

Country Status (19)

Country Link
US (2) US20220307020A1 (es)
EP (1) EP3956449B1 (es)
JP (1) JP7698828B2 (es)
KR (1) KR102851101B1 (es)
CN (1) CN113939591A (es)
AU (1) AU2020259548B2 (es)
BR (1) BR112021020608A8 (es)
CA (1) CA3136735A1 (es)
CL (1) CL2021002695A1 (es)
CO (1) CO2021015214A2 (es)
CR (1) CR20210572A (es)
EC (1) ECSP21080618A (es)
IL (1) IL287248A (es)
MX (1) MX2021012645A (es)
PE (1) PE20212214A1 (es)
PH (1) PH12021552613A1 (es)
SG (1) SG11202111401RA (es)
TW (1) TW202043249A (es)
WO (1) WO2020211780A1 (es)

Families Citing this family (38)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN109477103A (zh) 2016-06-22 2019-03-15 ProQR治疗上市公司Ⅱ 单链rna-编辑寡核苷酸
EP3507366B1 (en) 2016-09-01 2020-10-07 ProQR Therapeutics II B.V. Chemically modified single-stranded rna-editing oligonucleotides
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
TW202043249A (zh) 2019-04-15 2020-12-01 大陸商博雅輯因(北京)生物科技有限公司 編輯rna的方法和組合物
PH12022550059A1 (en) 2019-07-12 2022-11-21 Univ Beijing Targeted rna editing by leveraging endogenous adar using engineered rnas
CN115038789A (zh) 2019-12-02 2022-09-09 塑造治疗公司 治疗性编辑
WO2021136408A1 (zh) * 2019-12-30 2021-07-08 博雅辑因(北京)生物科技有限公司 一种基于leaper技术治疗mps ih的方法和组合物
TW202138561A (zh) * 2019-12-30 2021-10-16 大陸商博雅輯因(北京)生物科技有限公司 治療Usher綜合症的方法和其組合物
PH12022552687A1 (en) * 2020-04-15 2024-03-25 Edigene Therapeutics Beijing Inc Method and drug for treating hurler syndrome
CN113528582B (zh) * 2020-04-15 2022-05-17 博雅辑因(北京)生物科技有限公司 基于leaper技术靶向编辑rna的方法和药物
JP2023529316A (ja) * 2020-05-26 2023-07-10 シェイプ セラピューティクス インコーポレイテッド ゲノム編集のための組成物及び方法
WO2022150974A1 (en) * 2021-01-12 2022-07-21 Peking University Targeted rna editing by leveraging endogenous adar using engineered rnas
US20230194709A9 (en) * 2021-06-29 2023-06-22 Seagate Technology Llc Range information detection using coherent pulse sets with selected waveform characteristics
EP4388089A4 (en) * 2021-08-18 2025-01-15 Peking University Engineered adar-recruiting rnas and methods of use thereof
WO2023143539A1 (en) * 2022-01-28 2023-08-03 Edigene Therapeutics (Beijing) Inc. Engineered adar-recruiting rnas and methods of use thereof
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
US20260027237A1 (en) 2022-07-15 2026-01-29 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
JP2025536808A (ja) 2022-11-24 2025-11-07 プロキューアール・セラピューティクス・セカンド・ベスローテン・フェンノートシャップ 遺伝性hfeヘモクロマトーシスの治療のためのアンチセンスオリゴヌクレオチド
GB202218090D0 (en) 2022-12-01 2023-01-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency
JP2025540146A (ja) 2022-12-09 2025-12-11 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 心血管疾患の治療のためのアンチセンスオリゴヌクレオチド
GB202300865D0 (en) 2023-01-20 2023-03-08 Proqr Therapeutics Ii Bv Delivery of oligonucleotides
EP4669753A1 (en) 2023-02-20 2025-12-31 ProQR Therapeutics II B.V. Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease
WO2024206175A1 (en) 2023-03-24 2024-10-03 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
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
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
TW202546227A (zh) * 2024-01-18 2025-12-01 荷蘭商Proqr治療上市公司Ii 用於治療賀勒氏症候群(hurler syndrome)的反義寡核苷酸
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
CN121059628A (zh) * 2024-06-03 2025-12-05 时夕(广州)生物科技有限公司 调控RNA剪接的向导agRNA
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

Family Cites Families (41)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK187280A (da) 1980-04-30 1981-10-31 Novo Industri As Ruhedsreducerende middel til et fuldvaskemiddel fuldvaskemiddel og fuldvaskemetode
US5773244A (en) 1993-05-19 1998-06-30 Regents Of The University Of California Methods of making circular RNA
US9222088B2 (en) 2010-10-22 2015-12-29 Curna, Inc. Treatment of alpha-L-iduronidase (IDUA) related diseases by inhibition of natural antisense transcript to IDUA
US9650627B1 (en) 2012-07-19 2017-05-16 University Of Puerto Rico Site-directed RNA editing
EP3957735A1 (en) 2014-03-05 2022-02-23 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
EP4372091A3 (en) 2014-12-12 2024-07-31 Tod M. Woolf Compositions and methods for editing nucleic acids in cells utilizing oligonucleotides
CA2968336C (en) * 2014-12-17 2021-11-23 Proqr Therapeutics Ii B.V. Construct for site directed editing of an adenosine nucleotide in target rna
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
KR102368920B1 (ko) 2016-04-25 2022-02-28 프로큐알 테라퓨틱스 Ⅱ 비.브이. 안 질환 치료용 올리고뉴클레오타이드
CN109477103A (zh) * 2016-06-22 2019-03-15 ProQR治疗上市公司Ⅱ 单链rna-编辑寡核苷酸
WO2018041873A1 (en) 2016-09-01 2018-03-08 F. Hoffmann-La Roche Ag Process for the preparation of (6s)-6-isopropyl-10-methoxy-9-(3-methoxypropoxy)-2-oxo-6,7-dihydrobenzo[a]quinolizine-3-carboxylic acid
EP3507366B1 (en) 2016-09-01 2020-10-07 ProQR Therapeutics II B.V. Chemically modified single-stranded rna-editing oligonucleotides
GB201616202D0 (en) 2016-09-23 2016-11-09 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of eye deisease
EP3571300A1 (en) 2017-01-19 2019-11-27 ProQR Therapeutics II B.V. Oligonucleotide complexes for use in rna editing
EP3589751A4 (en) 2017-03-03 2021-11-17 The Regents of The University of California RNA TARGETING OF MUTATIONS VIA SUPPRESSOR RNA AND DEAMINASES
CA3059757A1 (en) * 2017-04-12 2018-10-18 Massachusetts Institute Of Technology Novel type vi crispr orthologs and systems
EP3622062A4 (en) 2017-05-10 2020-10-14 The Regents of the University of California DIRECTED EDITING OF CELLULAR RNA BY NUCLEAR ADMINISTRATION OF CRISPR / CAS9
WO2019005886A1 (en) 2017-06-26 2019-01-03 The Broad Institute, Inc. CRISPR / CAS-CYTIDINE DEAMINASE COMPOSITIONS, SYSTEMS AND METHODS FOR TARGETED EDITING OF NUCLEIC ACIDS
KR20200066616A (ko) 2017-09-21 2020-06-10 더 브로드 인스티튜트, 인코퍼레이티드 표적화된 핵산 편집을 위한 시스템, 방법 및 조성물
CA3111479A1 (en) 2017-09-26 2019-04-04 The Board Of Trustees Of The University Of Illinois Crispr/cas system and method for genome editing and modulating transcription
CA3077086A1 (en) 2017-10-04 2019-04-11 President And Fellows Of Harvard College Systems, methods, and compositions for targeted nucleic acid editing
AU2018393050A1 (en) 2017-12-21 2020-06-18 Bayer Healthcare Llc Materials and methods for treatment of Usher Syndrome Type 2A
EP3752611A1 (en) 2018-02-14 2020-12-23 ProQR Therapeutics II B.V. Antisense oligonucleotides for rna editing
EP4524247A3 (en) 2018-03-23 2025-06-11 Massachusetts Eye and Ear Infirmary Crispr/cas9-mediated exon-skipping approach for ush2a-associated usher syndrome
WO2020001793A1 (en) * 2018-06-29 2020-01-02 Eberhard-Karls-Universität Tübingen Artificial nucleic acids for rna editing
JP7720623B6 (ja) 2018-09-06 2025-09-18 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 遺伝子操作されたadarのリクルートを介したrna及びdna塩基の編集
KR102666695B1 (ko) * 2018-10-12 2024-05-23 페킹 유니버시티 Rna를 편집하기 위한 방법 및 조성물
CA3123981A1 (en) 2018-12-20 2020-06-25 Peking University Compositions and methods for highly efficient genetic screening using barcoded guide rna constructs
WO2020168051A1 (en) 2019-02-13 2020-08-20 Beam Therapeutics Inc. Methods of editing a disease-associated gene using adenosine deaminase base editors, including for the treatment of genetic disease
CN109943586B (zh) 2019-03-15 2021-02-26 上海交通大学 一种植物circRNA过表达载体及其构建方法
TW202043249A (zh) 2019-04-15 2020-12-01 大陸商博雅輯因(北京)生物科技有限公司 編輯rna的方法和組合物
CN113785059A (zh) 2019-04-30 2021-12-10 博雅辑因(北京)生物科技有限公司 一种血红蛋白病治疗有效性预测方法
PH12022550059A1 (en) 2019-07-12 2022-11-21 Univ Beijing Targeted rna editing by leveraging endogenous adar using engineered rnas
KR20220053671A (ko) 2019-09-04 2022-04-29 에디진 인크. 오프 타겟 평가 기반의 유전자 편집 치료의 평가 방법
US20230027247A1 (en) 2019-12-16 2023-01-26 Edigene (Guangzhou) Inc. Small molecule compounds for amplifying hematopoietic stem cells, and combination thereof
TW202138561A (zh) 2019-12-30 2021-10-16 大陸商博雅輯因(北京)生物科技有限公司 治療Usher綜合症的方法和其組合物
WO2021136408A1 (zh) 2019-12-30 2021-07-08 博雅辑因(北京)生物科技有限公司 一种基于leaper技术治疗mps ih的方法和组合物
JP2023509178A (ja) 2019-12-31 2023-03-07 北京▲輯▼因医▲療▼科技有限公司 Rnaをターゲティング編集する新しい方法
DE102020126779A1 (de) 2020-10-13 2022-04-14 Fritsch Bakery Technologies GmbH & Co. KG Teigverarbeitungsmaschine zum Bearbeiten von Produkten
WO2022150974A1 (en) 2021-01-12 2022-07-21 Peking University Targeted rna editing by leveraging endogenous adar using engineered rnas

Also Published As

Publication number Publication date
TW202043249A (zh) 2020-12-01
PE20212214A1 (es) 2021-11-19
EP3956449A1 (en) 2022-02-23
SG11202111401RA (en) 2021-11-29
AU2020259548A1 (en) 2021-12-09
KR102851101B1 (ko) 2025-08-27
US11702658B2 (en) 2023-07-18
EP3956449A4 (en) 2023-03-29
PH12021552613A1 (en) 2022-06-27
JP2022526455A (ja) 2022-05-24
CA3136735A1 (en) 2020-10-22
IL287248A (en) 2021-12-01
JP7698828B2 (ja) 2025-06-26
CL2021002695A1 (es) 2022-07-01
KR20220004674A (ko) 2022-01-11
US20220307020A1 (en) 2022-09-29
WO2020211780A1 (en) 2020-10-22
BR112021020608A8 (pt) 2023-03-21
EP3956449B1 (en) 2026-02-25
CR20210572A (es) 2022-04-07
CN113939591A (zh) 2022-01-14
US20220098587A1 (en) 2022-03-31
BR112021020608A2 (pt) 2022-02-22
CO2021015214A2 (es) 2022-02-07
AU2020259548B2 (en) 2023-10-12
MX2021012645A (es) 2021-11-12

Similar Documents

Publication Publication Date Title
ECSP21080618A (es) Methods and Compositions for Editing RNAs
ECSP21033637A (es) Methods and Compositions for Editing RNAs
CO2022001359A2 (es) Edición de ácido ribonucleico (arn) dirigido aprovechando la adenosina desaminasa que actúa sobre ácido ribonucleico endógeno (adar) utilizando ácidos ribonucleicos (arn) modificados genéticamente
MX2019003674A (es) Enzimas modificadoras de ácido nucleico guiadas por arn y métodos de uso de estas.
BR102016018569A2 (pt) sistemas e métodos de retentor para eletrodo de soldagem com bastão
CO2018004763A2 (es) Edición genómica multiplexada
BR112018076190A2 (pt) ortólogos e sistemas crispr tipo vi
GB2568182A (en) Adenosine nucleobase editors and uses thereof
BR112017016045A2 (pt) método para induzir recombinações meióticas endereçadas em uma célula eucariótica, proteína de fusão, ácido nucleico, cassete de expressão ou um vetor, célula hospedeira, método para gerar variantes de um organismo eucariótico, método para identificar ou localizar as informações genéticas, kit, e, uso de um kit
MX2016004575A (es) Metodos de modificacion de celulas huespedes.
MX2022001085A (es) Anticuerpos anti-c1s humanizados y metodos para usarlos.
BR112015030946A8 (pt) composição compreendendo uma célula ß pancreática não-nativa, método de diferenciação de células progenitoras em células ß pancreáticas in vitro e uso de uma célula ß pancreática não-nativa
BR112022017196A2 (pt) Recombinação genômica guiada por rna na escala de kilobase
AR110871A1 (es) Proteínas de fusión de factor ix y métodos de fabricación y uso de las mismas
CL2019000920A1 (es) Métodos de preservar la actividad biológica de los ácidos ribonucleicos.
EA202191004A1 (ru) Способы и композиции для редактирования рнк
CL2021001708A1 (es) Métodos para formar poliplejos
CR20240112A (es) Compuestos y métodos para modular splicing
MX2021013525A (es) Metodos y composiciones que usan celulas regulables auxotroficas.
CL2018000872A1 (es) Métodos de preservar la actividad biológica de los ácidos ribonucleicos.
AR115486A1 (es) Nucleasas guiadas por arn y sus fragmentos activos y variantes y métodos de uso
AR120928A1 (es) Nucleasas guiadas por arn y sus fragmentos activos y variantes y métodos de uso
BR112018015626A2 (pt) sistema fúngico de produção de alto nível de proteínas