CL2022000080A1 - Edición de ácido ribonucleico (arn) dirigido aprovechando la adenosina desaminasa que actúa sobre ácido ribonucleico endógeno (adar) utilizando ácidos ribonucleicos (arn) modificados genéticamente - Google Patents

Edición de ácido ribonucleico (arn) dirigido aprovechando la adenosina desaminasa que actúa sobre ácido ribonucleico endógeno (adar) utilizando ácidos ribonucleicos (arn) modificados genéticamente

Info

Publication number
CL2022000080A1
CL2022000080A1 CL2022000080A CL2022000080A CL2022000080A1 CL 2022000080 A1 CL2022000080 A1 CL 2022000080A1 CL 2022000080 A CL2022000080 A CL 2022000080A CL 2022000080 A CL2022000080 A CL 2022000080A CL 2022000080 A1 CL2022000080 A1 CL 2022000080A1
Authority
CL
Chile
Prior art keywords
rna
ribonucleic acid
ribonucleic
adar
endogenous
Prior art date
Application number
CL2022000080A
Other languages
English (en)
Inventor
Wensheng Wei
Liang Qu
Zongyi Yi
Shiyou Zhu
Chunhui Wang
Zhuo Zhou
Feng Tian
Original Assignee
Univ Beijing
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 filed Critical Univ Beijing
Publication of CL2022000080A1 publication Critical patent/CL2022000080A1/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
    • C12N15/63Introduction 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
    • C12N15/10Processes 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
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • 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
    • 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
    • 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
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • 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
    • 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
    • C12N2310/12Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • 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
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/532Closed or circular
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/34Allele or polymorphism specific uses
    • 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
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/16011Herpesviridae
    • C12N2710/16111Cytomegalovirus, e.g. human herpesvirus 5
    • C12N2710/16141Use of virus, viral particle or viral elements as a vector
    • C12N2710/16145Special targeting system for viral vectors
    • 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
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/16011Human Immunodeficiency Virus, HIV
    • C12N2740/16041Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Virology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (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 y kits que comprenden los mismos.
CL2022000080A 2019-07-12 2022-01-11 Edición de ácido ribonucleico (arn) dirigido aprovechando la adenosina desaminasa que actúa sobre ácido ribonucleico endógeno (adar) utilizando ácidos ribonucleicos (arn) modificados genéticamente CL2022000080A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
CN2019095802 2019-07-12

Publications (1)

Publication Number Publication Date
CL2022000080A1 true CL2022000080A1 (es) 2022-11-18

Family

ID=74210191

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2022000080A CL2022000080A1 (es) 2019-07-12 2022-01-11 Edición de ácido ribonucleico (arn) dirigido aprovechando la adenosina desaminasa que actúa sobre ácido ribonucleico endógeno (adar) utilizando ácidos ribonucleicos (arn) modificados genéticamente

Country Status (15)

Country Link
US (3) US20220243194A1 (es)
EP (1) EP3997229A1 (es)
JP (1) JP2022536546A (es)
KR (1) KR20220038706A (es)
CN (2) CN117778385A (es)
AU (1) AU2020313143B2 (es)
CA (1) CA3146771A1 (es)
CL (1) CL2022000080A1 (es)
CO (1) CO2022001359A2 (es)
CR (1) CR20220063A (es)
EC (1) ECSP22010372A (es)
IL (1) IL289700A (es)
MX (1) MX2022000497A (es)
PE (1) PE20220300A1 (es)
WO (1) WO2021008447A1 (es)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2020259548B2 (en) 2019-04-15 2023-10-12 Edigene Therapeutics (Beijing) Inc. Methods and compositions for editing RNAs
CR20220063A (es) 2019-07-12 2022-07-22 Univ Beijing Edición de ácido ribonucleico (arn) dirigido aprovechando la adenosina desaminasa que actúa sobre ácido ribonucleico endógeno (adar) utilizando ácidos ribonucleicos (arn) modificados genéticamente
JP2023504314A (ja) 2019-12-02 2023-02-02 シェイプ セラピューティクス インコーポレイテッド 治療的編集
PE20230703A1 (es) * 2020-04-15 2023-04-24 Edigene Therapeutics Beijing Inc Metodo y farmaco para tratar el sindrome de hurler
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
WO2022256578A2 (en) * 2021-06-02 2022-12-08 Beam Therapeutics Inc. Circular guide rnas for crispr/cas editing systems
WO2023020574A1 (en) * 2021-08-18 2023-02-23 Peking University Engineered adar-recruiting rnas and methods of use thereof
WO2023143538A1 (zh) * 2022-01-28 2023-08-03 北京辑因医疗科技有限公司 基于leaper技术治疗mpsi的方法和组合物
WO2023152371A1 (en) 2022-02-14 2023-08-17 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
WO2023237063A1 (en) * 2022-06-08 2023-12-14 Huidagene Therapeutics Co., Ltd. Novel guide nucleic acids for rna base editing systems and uses thereof
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
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
GB202218090D0 (en) 2022-12-01 2023-01-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency
WO2024121373A1 (en) 2022-12-09 2024-06-13 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of cardiovascular disease

Family Cites Families (40)

* 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
RU2597972C2 (ru) 2010-10-22 2016-09-20 Курна Инк. Лечение заболеваний, связанных с геном альфа-l-идуронидазы (idua), путем ингибирования природного антисмыслового транскрипта гена 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
CN107532162A (zh) 2014-12-12 2018-01-02 托德·M·伍尔夫 用于利用寡核苷酸编辑细胞中核酸的组合物和方法
WO2016097212A1 (en) * 2014-12-17 2016-06-23 Proqr Therapeutics Ii B.V. Targeted rna editing
EP3323890A4 (en) 2015-07-14 2019-01-30 Fukuoka University METHOD FOR INDUCING SITE SPECIFIC RNA MUTATIONS, TARGET EDITION GUIDING RNA-GUIDE USED IN THE METHOD, AND TARGET EDITING GUID-RNA TARGET RNA COMPLEX
DE102015012522B3 (de) 2015-09-26 2016-06-02 Eberhard Karls Universität Tübingen Verfahren und Substanzen zur gerichteten RNA-Editierung
ES2801823T3 (es) 2016-04-25 2021-01-14 Proqr Therapeutics Ii Bv Oligonucleótidos para tratar una enfermedad ocular
CA3024944A1 (en) 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Single-stranded rna-editing oligonucleotides
AU2017320901B2 (en) 2016-09-01 2023-11-09 Proqr Therapeutics Ii B.V. Chemically modified single-stranded RNA-editing oligonucleotides
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
GB201616202D0 (en) 2016-09-23 2016-11-09 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of eye deisease
US11274300B2 (en) 2017-01-19 2022-03-15 Proqr Therapeutics Ii B.V. Oligonucleotide complexes for use in RNA editing
WO2018161032A1 (en) * 2017-03-03 2018-09-07 The Regents Of The University Of California RNA TARGETING OF MUTATIONS VIA SUPPRESSOR tRNAs AND DEAMINASES
CN110869498A (zh) 2017-05-10 2020-03-06 加利福尼亚大学董事会 经由核递送crispr/cas9导向编辑细胞rna
US20200248169A1 (en) 2017-06-26 2020-08-06 The Broad Institute, Inc. Crispr/cas-cytidine deaminase based compositions, systems, and methods for targeted nucleic acid editing
US11618896B2 (en) 2017-09-21 2023-04-04 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
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
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
CA3084632A1 (en) 2017-12-21 2019-06-27 Crispr Therapeutics Ag Materials and methods for treatment of usher syndrome type 2a
US20210079393A1 (en) 2018-02-14 2021-03-18 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for rna editing
EP3768303A4 (en) 2018-03-23 2021-12-29 Massachusetts Eye and Ear Infirmary Crispr/cas9-mediated exon-skipping approach for ush2a-associated usher syndrome
CN112752844A (zh) 2018-06-29 2021-05-04 蒂宾根大学 用于rna编辑的人工核酸
GB2590880A (en) * 2018-09-06 2021-07-07 Univ California RNA and DNA base editing via engineered ADAR recruitment
CN116218836A (zh) 2018-10-12 2023-06-06 北京大学 编辑rna的方法和组合物
AU2019408503B2 (en) 2018-12-20 2023-06-29 Edigene Biotechnology Inc. Compositions and methods for highly efficient genetic screening using barcoded guide rna constructs
JP2022520080A (ja) 2019-02-13 2022-03-28 ビーム セラピューティクス インク. 遺伝的疾患の治療用を含めアデノシンデアミナーゼ塩基エディターを用いて疾患関連遺伝子を編集する方法
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
CN113939617A (zh) 2019-03-26 2022-01-14 北京大学 鉴定功能元件的方法
AU2020259548B2 (en) 2019-04-15 2023-10-12 Edigene Therapeutics (Beijing) Inc. Methods and compositions for editing RNAs
KR20220002565A (ko) 2019-04-30 2022-01-06 에디진 인크. 이상헤모글로빈증 치료의 효과를 예측하는 방법
CR20220063A (es) 2019-07-12 2022-07-22 Univ Beijing Edición de ácido ribonucleico (arn) dirigido aprovechando la adenosina desaminasa que actúa sobre ácido ribonucleico endógeno (adar) utilizando ácidos ribonucleicos (arn) modificados genéticamente
CN114269941A (zh) 2019-09-04 2022-04-01 博雅辑因(北京)生物科技有限公司 基于脱靶评估评价基因编辑治疗的方法
TW202136503A (zh) 2019-12-16 2021-10-01 大陸商廣州輯因醫療科技有限公司 擴增造血幹細胞的小分子化合物及其組合
EP4086346A4 (en) 2019-12-30 2024-03-20 EdiGene Therapeutics (Beijing) Inc. METHOD FOR TREATMENT OF USHER SYNDROME AND ASSOCIATED COMPOSITION
PE20230037A1 (es) 2019-12-30 2023-01-10 Edigene Therapeutics Beijing Inc Metodo basado en tecnologia leaper para el tratamiento de mucopolisacaridosis ih y composicion
EP4085931A4 (en) 2019-12-31 2024-01-17 EdiGene Therapeutics (Beijing) Inc. NEW PROCESS FOR TARGETED RNA EDITING
JP2024504608A (ja) 2021-01-12 2024-02-01 北京大学 遺伝子操作rnaを使用した内因性adarの活用による標的化rnaの編集

Also Published As

Publication number Publication date
EP3997229A1 (en) 2022-05-18
JP2022536546A (ja) 2022-08-17
US11661596B2 (en) 2023-05-30
AU2020313143A1 (en) 2022-02-10
AU2020313143B2 (en) 2024-04-04
CR20220063A (es) 2022-07-22
CN114269919B (zh) 2023-08-18
IL289700A (en) 2022-03-01
MX2022000497A (es) 2022-02-03
CN114269919A (zh) 2022-04-01
CO2022001359A2 (es) 2022-05-10
PE20220300A1 (es) 2022-03-09
BR112022000291A2 (pt) 2022-03-15
KR20220038706A (ko) 2022-03-29
ECSP22010372A (es) 2022-03-31
WO2021008447A1 (en) 2021-01-21
US20220135963A1 (en) 2022-05-05
CA3146771A1 (en) 2021-01-21
US20220243194A1 (en) 2022-08-04
US20230365962A1 (en) 2023-11-16
CN117778385A (zh) 2024-03-29

Similar Documents

Publication Publication Date Title
CL2022000080A1 (es) Edición de ácido ribonucleico (arn) dirigido aprovechando la adenosina desaminasa que actúa sobre ácido ribonucleico endógeno (adar) utilizando ácidos ribonucleicos (arn) modificados genéticamente
CO2021005956A2 (es) Métodos y composiciones para editar ácidos ribonucleicos
CO2021015214A2 (es) Métodos y composiciones para editar ácidos ribonucleicos (arn)
CO2018004763A2 (es) Edición genómica multiplexada
BR112017011510A2 (pt) edição de rna direcionado
CO2022010430A2 (es) Método para el tratamiento del síndrome de usher y composición del mismo
BRPI1007214A2 (pt) adaptador para sequenciar ácidos nucleicos, par de adaptadores, kit, construto de ácido nucleico, e, métodos para preparar um adaptador, um construto de ácido nucleico, e um construto de dequência, e para sequenciar ácido nucleico de fita dupla.
EA201291024A1 (ru) Композиции эндорибонуклеаз и способы их использования
BR112015022491A2 (pt) variantes de inibidor de tecido de metaloprotease tipo três (timp-3), ácido nucleico isolado, vetor de expressão, célula hospedeira isolada e método para produzir uma muteína de timp-3 recombinante, bem como composição e seu uso
ES2570382T3 (es) Composiciones y métodos para la producción de azúcares fermentables
MX2020013384A (es) Compuestos de comarina sustituidos con amina exocíclica y sus usos como etiquetas fluorescentes.
BR112022020795A2 (pt) Métodos para edição alvejada de um ácido ribonucleico alvo e para prevenir ou tratar síndrome de hurler, ácido ribonucleico de recrutamento da adenosina desaminase engenheirado, construção, e, composição, lipossoma, exossoma, nanopartícula de lipídeo, célula, biblioteca ou kit
BR112022002695A2 (pt) Nucleases guiadas por rna, fragmentos ativos e variantes das mesmas e métodos de uso
WO2009093242A3 (en) Cell populations for polypeptide analysis and uses of same
BR112022011426A2 (pt) Uso de inibidores de atr em combinação com inibidores de parp
BR112014022467A2 (pt) transformantes de rasamsonia
BR112022017196A2 (pt) Recombinação genômica guiada por rna na escala de kilobase
CY1114586T1 (el) Συνθεση φορεα για τη μεταφορα νουκλεϊνικων οξεων
ES2461896B1 (es) Métodos para obtener plantas resistentes a sequía
CO6341568A2 (es) Composicion inmunologica
AR065847A1 (es) Composicion portadora de rapida accion para la insercion de acido nucleico. metodo. uso.
EA202191004A1 (ru) Способы и композиции для редактирования рнк
CL2020001651A1 (es) Modificación dirigida inducible de ácidos nucleicos por luz e información genética
AR106259A1 (es) Métodos de preservar la actividad biológica de los ácidos ribonucleicos
PH12019500738A1 (en) Methods of preserving the biological activity of ribonucleic acids