CL2021000880A1 - Métodos y composiciones para editar arn - Google Patents

Métodos y composiciones para editar arn

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Publication number
CL2021000880A1
CL2021000880A1 CL2021000880A CL2021000880A CL2021000880A1 CL 2021000880 A1 CL2021000880 A1 CL 2021000880A1 CL 2021000880 A CL2021000880 A CL 2021000880A CL 2021000880 A CL2021000880 A CL 2021000880A CL 2021000880 A1 CL2021000880 A1 CL 2021000880A1
Authority
CL
Chile
Prior art keywords
methods
compositions
arn
edit
rna
Prior art date
Application number
CL2021000880A
Other languages
English (en)
Inventor
Wensheng Wei
Liang Qu
Zongyi Yi
Shiyou Zhu
Chunhui Wang
Zhongzheng Cao
Zhuo Zhou
Pengfei Yuan
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 CL2021000880A1 publication Critical patent/CL2021000880A1/es

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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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    • 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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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
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  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • General Chemical & Material Sciences (AREA)
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  • Cardiology (AREA)
  • Hematology (AREA)
  • Cell Biology (AREA)
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  • Hospice & Palliative Care (AREA)
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Abstract

Se proporcionan métodos para editar ARN introduciendo un ARN reclutador de deaminasa en una célula huésped para la desaminación de una adenosina en un ARN diana, y ARN reclutadores de deaminasa utilizados en los métodos de edición de ARN y composiciones que comprenden los mismos.
CL2021000880A 2018-10-12 2021-04-09 Métodos y composiciones para editar arn CL2021000880A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
CN2018110105 2018-10-12
CN2019082713 2019-04-15

Publications (1)

Publication Number Publication Date
CL2021000880A1 true CL2021000880A1 (es) 2021-12-10

Family

ID=70163607

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2021000880A CL2021000880A1 (es) 2018-10-12 2021-04-09 Métodos y composiciones para editar arn

Country Status (20)

Country Link
US (2) US20210355494A1 (es)
EP (2) EP3864152B1 (es)
JP (2) JP7252328B2 (es)
CN (4) CN113631708B (es)
AU (2) AU2019357450B2 (es)
BR (1) BR112021006844A8 (es)
CA (1) CA3115864A1 (es)
CL (1) CL2021000880A1 (es)
CO (1) CO2021005956A2 (es)
CR (1) CR20210243A (es)
DK (1) DK3864152T3 (es)
EC (1) ECSP21033637A (es)
ES (1) ES2962434T3 (es)
IL (1) IL282191A (es)
MX (1) MX2021004187A (es)
PE (1) PE20211402A1 (es)
PH (1) PH12021550805A1 (es)
SG (1) SG11202103666PA (es)
TW (1) TW202028466A (es)
WO (1) WO2020074001A1 (es)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2020508685A (ja) 2017-03-03 2020-03-26 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 サプレッサーｔＲＮＡ及びデアミナーゼによる変異のＲＮＡターゲティング
US20220307020A1 (en) 2019-04-15 2022-09-29 Edigene Inc. Methods and compositions for editing rnas
CN117778385A (zh) 2019-07-12 2024-03-29 北京大学 采用工程化rna利用内源adar进行靶向的rna编辑
CN115038789A (zh) 2019-12-02 2022-09-09 塑造治疗公司 治疗性编辑
CA3163272A1 (en) 2019-12-30 2021-07-08 Pengfei YUAN Leaper technology based method for treating mps ih and composition
IL294260A (en) 2019-12-30 2022-08-01 Edigene Therapeutics Beijing Inc A method and preparation for the treatment of Asher syndrome
KR20220162168A (ko) * 2020-04-15 2022-12-07 에디진 테라퓨틱스 (베이징) 인크. 헐러 증후군의 치료 방법 및 약물
EP4157359A1 (en) * 2020-05-28 2023-04-05 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of serpina1
CN113897359A (zh) * 2020-07-06 2022-01-07 博雅辑因（北京）生物科技有限公司 一种改善的rna编辑方法
AU2021305359A1 (en) 2020-07-06 2023-02-16 Edigene Therapeutics (Beijing) Inc. Improved RNA editing method
IL278401A (en) 2020-10-29 2022-05-01 Yeda Res & Dev Polynucleotides for editing RNA and a method for using them
WO2022147573A1 (en) * 2021-01-04 2022-07-07 The Regents Of The University Of California Programmable rna editing in vivo via recruitment of endogenous adars
CN117015605A (zh) * 2021-01-12 2023-11-07 北京大学 使用工程化rna通过利用内源性adar进行靶向rna编辑
KR20240021893A (ko) * 2021-06-15 2024-02-19 메사추세츠 인스티튜트 오브 테크놀로지 데아미나제 기반 rna 센서
WO2023278436A1 (en) * 2021-06-29 2023-01-05 Shape Therapeutics Inc. Engineered guide rnas and polynucleotides
WO2023020574A1 (en) * 2021-08-18 2023-02-23 Peking University Engineered adar-recruiting rnas and methods of use thereof
TW202321440A (zh) 2021-10-07 2023-06-01 美商肝特斯公司 追蹤接受者中之供體細胞之方法
AU2022377076A1 (en) * 2021-10-29 2024-05-02 Cold Spring Harbor Laboratory Compositions and systems for rna-programmable cell editing and methods of making and using same
WO2023143539A1 (en) * 2022-01-28 2023-08-03 Edigene Therapeutics (Beijing) Inc. Engineered adar-recruiting rnas and methods of use thereof
WO2023185231A1 (en) * 2022-04-02 2023-10-05 Edigene Therapeutics (Beijing) Inc. Engineered adar-recruiting rnas and methods of use for usher syndrome
WO2024086650A1 (en) * 2022-10-18 2024-04-25 Locanabio, Inc. Compositions and methods comprising programmable snrnas for rna editing

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3230445B1 (en) 2014-12-12 2024-01-24 Tod M. Woolf Compositions and methods for editing nucleic acids in cells utilizing oligonucleotides
EP3712269A1 (en) 2014-12-17 2020-09-23 ProQR Therapeutics II B.V. Targeted rna editing
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
JP7074345B2 (ja) * 2016-06-22 2022-05-24 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 一本鎖ｒｎａ編集オリゴヌクレオチド
EP3507366B1 (en) * 2016-09-01 2020-10-07 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
JP2020508685A (ja) * 2017-03-03 2020-03-26 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 サプレッサーｔＲＮＡ及びデアミナーゼによる変異のＲＮＡターゲティング
WO2018170333A1 (en) 2017-03-15 2018-09-20 The Broad Institute, Inc. Novel cas13b orthologues crispr enzymes and systems

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US20210355494A1 (en) 2021-11-18
CA3115864A1 (en) 2020-04-16
JP2023078373A (ja) 2023-06-06
BR112021006844A8 (pt) 2023-03-21
CO2021005956A2 (es) 2021-07-30
EP3864152B1 (en) 2023-08-16
EP4306116A3 (en) 2024-04-17
MX2021004187A (es) 2021-09-08
ES2962434T3 (es) 2024-03-19
CN113631708A (zh) 2021-11-09
EP3864152A4 (en) 2021-10-13
BR112021006844A2 (pt) 2021-07-20
DK3864152T3 (da) 2023-10-09
IL282191A (en) 2021-05-31
CN116042611A (zh) 2023-05-02
CN115651927B (zh) 2023-06-16
SG11202103666PA (en) 2021-05-28
AU2023202184A1 (en) 2023-05-04
AU2019357450B2 (en) 2023-01-12
KR20210076082A (ko) 2021-06-23
ECSP21033637A (es) 2021-08-31
JP2021534810A (ja) 2021-12-16
EP4306116A2 (en) 2024-01-17
EP3864152A1 (en) 2021-08-18
CN115651927A (zh) 2023-01-31
CN116218836A (zh) 2023-06-06
TW202028466A (zh) 2020-08-01
JP7252328B2 (ja) 2023-04-04
CN113631708B (zh) 2022-05-24
AU2019357450A1 (en) 2021-06-03
PH12021550805A1 (en) 2021-10-04
WO2020074001A1 (en) 2020-04-16
US20210310026A1 (en) 2021-10-07
CR20210243A (es) 2021-10-07
PE20211402A1 (es) 2021-08-02

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