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 genéticamenteInfo
- 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
Links
- 229920002477 rna polymer Polymers 0.000 title 5
- 101710169336 5'-deoxyadenosine deaminase Proteins 0.000 title 1
- 102000055025 Adenosine deaminases Human genes 0.000 title 1
- OIRDTQYFTABQOQ-KQYNXXCUSA-N adenosine Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O OIRDTQYFTABQOQ-KQYNXXCUSA-N 0.000 abstract 2
- 238000000034 method Methods 0.000 abstract 2
- 239000002126 C01EB10 - Adenosine Substances 0.000 abstract 1
- 238000010357 RNA editing Methods 0.000 abstract 1
- 230000026279 RNA modification Effects 0.000 abstract 1
- 229960005305 adenosine Drugs 0.000 abstract 1
- 230000009615 deamination Effects 0.000 abstract 1
- 238000006481 deamination reaction Methods 0.000 abstract 1
- 230000014789 establishment of RNA localization Effects 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 230000007115 recruitment Effects 0.000 abstract 1
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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
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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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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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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
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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
- C12N15/11—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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- 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
- C12N15/86—Viral vectors
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
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- C12Y—ENZYMES
- C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
- C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
- C12Y305/04004—Adenosine deaminase (3.5.4.4)
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
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- C12N2310/3519—Fusion with another nucleic acid
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/532—Closed or circular
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/34—Allele or polymorphism specific uses
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- C12N2710/00011—Details
- C12N2710/16011—Herpesviridae
- C12N2710/16111—Cytomegalovirus, e.g. human herpesvirus 5
- C12N2710/16141—Use of virus, viral particle or viral elements as a vector
- C12N2710/16145—Special targeting system for viral vectors
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- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/16011—Human Immunodeficiency Virus, HIV
- C12N2740/16041—Use of virus, viral particle or viral elements as a vector
- C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
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- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
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- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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- 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.
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
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CN2019095802 | 2019-07-12 |
Publications (1)
Publication Number | Publication Date |
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CL2022000080A1 true CL2022000080A1 (es) | 2022-11-18 |
Family
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Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
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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 |
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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) |
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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 |
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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 |
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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 |
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JP2022536546A (ja) | 2022-08-17 |
US11661596B2 (en) | 2023-05-30 |
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AU2020313143B2 (en) | 2024-04-04 |
CR20220063A (es) | 2022-07-22 |
CN114269919B (zh) | 2023-08-18 |
IL289700A (en) | 2022-03-01 |
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CN114269919A (zh) | 2022-04-01 |
CO2022001359A2 (es) | 2022-05-10 |
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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 |
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