PE20211402A1 - Metodos y composiciones para editar acidos ribonucleicos - Google Patents

Metodos y composiciones para editar acidos ribonucleicos

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Publication number
PE20211402A1
PE20211402A1 PE2021000498A PE2021000498A PE20211402A1 PE 20211402 A1 PE20211402 A1 PE 20211402A1 PE 2021000498 A PE2021000498 A PE 2021000498A PE 2021000498 A PE2021000498 A PE 2021000498A PE 20211402 A1 PE20211402 A1 PE 20211402A1
Authority
PE
Peru
Prior art keywords
editing
methods
rna
compositions
ribonucleic acids
Prior art date
Application number
PE2021000498A
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 PE20211402A1 publication Critical patent/PE20211402A1/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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A61K31/7105—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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
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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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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • 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
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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)
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Abstract

La presente solicitud proporciona metodos para editar ARN mediante la introduccion de un ARN de reclutamiento de desaminasas en una celula hospedera para la desaminacion de una adenosina en un ARN objetivo. La presente solicitud proporciona ademas ARN de reclutamiento de desaminasas usados en los metodos de edicion de ARN y composiciones que los comprenden
PE2021000498A 2018-10-12 2019-10-12 Metodos y composiciones para editar acidos ribonucleicos PE20211402A1 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
CN2018110105 2018-10-12
CN2019082713 2019-04-15
PCT/CN2019/110782 WO2020074001A1 (en) 2018-10-12 2019-10-12 Methods and Compositions for Editing RNAs

Publications (1)

Publication Number Publication Date
PE20211402A1 true PE20211402A1 (es) 2021-08-02

Family

ID=70163607

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2021000498A PE20211402A1 (es) 2018-10-12 2019-10-12 Metodos y composiciones para editar acidos ribonucleicos

Country Status (20)

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US (2) US20210355494A1 (es)
EP (2) EP4306116A3 (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
US11932856B2 (en) 2017-03-03 2024-03-19 The Regents Of The University Of California RNA targeting of mutations via suppressor tRNAs and deaminases
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
MX2022000497A (es) 2019-07-12 2022-02-03 Univ Beijing Edicion de acido ribonucleico (arn) dirigido aprovechando la adenosina desaminasa que actua sobre acido ribonucleico endogeno (adar) utilizando acidos ribonucleicos (arn) modificados geneticamente.
CN115038789A (zh) 2019-12-02 2022-09-09 塑造治疗公司 治疗性编辑
JP2023509179A (ja) 2019-12-30 2023-03-07 北京▲輯▼因医▲療▼科技有限公司 Ｌｅａｐｅｒ技術に基づくｍｐｓ ｉｈの治療方法及び組成物
BR112022012921A2 (pt) 2019-12-30 2022-09-06 Edigene Therapeutics Beijing Inc Métodos para editar de forma alvejada ácido ribonucleico e para tratar a síndrome de usher tipo ii, ácido ribonucleico de recrutamento da adenosina desaminase, construto ou vetor de entrega, célula, e, uso do ácido ribonucleico de recrutamento da adenosina desaminase ou do construto ou vetor de entrega
EP4137161A4 (en) * 2020-04-15 2023-10-11 EdiGene Therapeutics (Beijing) Inc. METHOD AND MEDICATION FOR THE TREATMENT OF HURLER SYNDROME
KR20230033651A (ko) * 2020-05-28 2023-03-08 코로 바이오, 인크. Serpina1의 adar-매개 편집을 위한 방법 및 조성물
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
WO2022007803A1 (zh) 2020-07-06 2022-01-13 博雅辑因（北京）生物科技有限公司 一种改善的rna编辑方法
IL278401A (en) 2020-10-29 2022-05-01 Yeda Res & Dev Polynucleotides for editing RNA and a method for using them
EP4240338A1 (en) * 2021-01-04 2023-09-13 The Regents of The University of California <smallcaps/>? ? ?in vivo? ? ? ? ?programmable rna editingvia recruitment of endogenous adars
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
US20230123513A1 (en) * 2021-06-15 2023-04-20 Massachusetts Institute Of Technology Deaminase-based rna sensors
EP4363576A1 (en) * 2021-06-29 2024-05-08 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
WO2023060186A1 (en) 2021-10-07 2023-04-13 Hepatx Corporation Methods of tracking donor cells in a recipient
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
CA2969464A1 (en) * 2014-12-12 2016-06-16 Tod M. Woolf Compositions and methods for editing nucleic acids in cells utilizing oligonucleotides
PL3234134T3 (pl) * 2014-12-17 2020-12-28 Proqr Therapeutics Ii B.V. Ukierunkowana edycja rna
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
AU2017281497B2 (en) * 2016-06-22 2023-04-06 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
US11274300B2 (en) * 2017-01-19 2022-03-15 Proqr Therapeutics Ii B.V. Oligonucleotide complexes for use in RNA editing
US11932856B2 (en) * 2017-03-03 2024-03-19 The Regents Of The University Of California RNA targeting of mutations via suppressor tRNAs and deaminases
CN110959039A (zh) * 2017-03-15 2020-04-03 博德研究所 新型cas13b直向同源物crispr酶和系统

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Publication number Publication date
EP3864152B1 (en) 2023-08-16
US20210355494A1 (en) 2021-11-18
CA3115864A1 (en) 2020-04-16
JP2023078373A (ja) 2023-06-06
CN115651927B (zh) 2023-06-16
BR112021006844A8 (pt) 2023-03-21
US20210310026A1 (en) 2021-10-07
CN113631708B (zh) 2022-05-24
KR20210076082A (ko) 2021-06-23
DK3864152T3 (da) 2023-10-09
CO2021005956A2 (es) 2021-07-30
AU2019357450B2 (en) 2023-01-12
EP4306116A3 (en) 2024-04-17
JP2021534810A (ja) 2021-12-16
CN113631708A (zh) 2021-11-09
TW202028466A (zh) 2020-08-01
ECSP21033637A (es) 2021-08-31
ES2962434T3 (es) 2024-03-19
CN116218836A (zh) 2023-06-06
BR112021006844A2 (pt) 2021-07-20
IL282191A (en) 2021-05-31
AU2019357450A1 (en) 2021-06-03
EP4306116A2 (en) 2024-01-17
EP3864152A1 (en) 2021-08-18
CN116042611A (zh) 2023-05-02
JP7252328B2 (ja) 2023-04-04
CR20210243A (es) 2021-10-07
SG11202103666PA (en) 2021-05-28
PH12021550805A1 (en) 2021-10-04
CL2021000880A1 (es) 2021-12-10
EP3864152A4 (en) 2021-10-13
CN115651927A (zh) 2023-01-31
AU2023202184A1 (en) 2023-05-04
MX2021004187A (es) 2021-09-08
WO2020074001A1 (en) 2020-04-16

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