MX2022000497A - Edicion de acido ribonucleico (arn) dirigido aprovechando la adenosina desaminasa que actua sobre acido ribonucleico endogeno (adar) utilizando acidos ribonucleicos (arn) modificados geneticamente. - Google Patents
Edicion de acido ribonucleico (arn) dirigido aprovechando la adenosina desaminasa que actua sobre acido ribonucleico endogeno (adar) utilizando acidos ribonucleicos (arn) modificados geneticamente.Info
- Publication number
- MX2022000497A MX2022000497A MX2022000497A MX2022000497A MX2022000497A MX 2022000497 A MX2022000497 A MX 2022000497A MX 2022000497 A MX2022000497 A MX 2022000497A MX 2022000497 A MX2022000497 A MX 2022000497A MX 2022000497 A MX2022000497 A MX 2022000497A
- Authority
- MX
- Mexico
- Prior art keywords
- rna editing
- targeted rna
- endogenous adar
- leveraging
- rna
- Prior art date
Links
- 108091032973 (ribonucleotides)n+m Proteins 0.000 title abstract 6
- 102000040650 (ribonucleotides)n+m Human genes 0.000 title abstract 2
- 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
- 239000000203 mixture Substances 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
- C12N15/09—Recombinant DNA-technology
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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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- 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
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- C12N2310/11—Antisense
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- C12N2310/00—Structure or type of the nucleic acid
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- 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
- C12N2310/351—Conjugate
- 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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- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- 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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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Medicinal Chemistry (AREA)
- Crystallography & Structural Chemistry (AREA)
- Epidemiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Virology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Enzymes And Modification Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (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.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
CN2019095802 | 2019-07-12 | ||
PCT/CN2020/101246 WO2021008447A1 (en) | 2019-07-12 | 2020-07-10 | Targeted rna editing by leveraging endogenous adar using engineered rnas |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2022000497A true MX2022000497A (es) | 2022-02-03 |
Family
ID=74210191
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2022000497A MX2022000497A (es) | 2019-07-12 | 2020-07-10 | Edicion de acido ribonucleico (arn) dirigido aprovechando la adenosina desaminasa que actua sobre acido ribonucleico endogeno (adar) utilizando acidos ribonucleicos (arn) modificados geneticamente. |
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 (13)
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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 |
KR20220162168A (ko) * | 2020-04-15 | 2022-12-07 | 에디진 테라퓨틱스 (베이징) 인크. | 헐러 증후군의 치료 방법 및 약물 |
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 |
AU2022285744A1 (en) * | 2021-06-02 | 2023-12-14 | 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 |
WO2024013361A1 (en) | 2022-07-15 | 2024-01-18 | Proqr Therapeutics Ii B.V. | Oligonucleotides for adar-mediated rna editing and use thereof |
WO2024013360A1 (en) | 2022-07-15 | 2024-01-18 | 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 |
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KR20220116508A (ko) | 2019-12-16 | 2022-08-23 | 에디진 (광저우) 인크. | 조혈 줄기 세포의 증폭을 위한 저분자 화합물 및 이의 조합 |
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 |
AU2020417930A1 (en) | 2019-12-31 | 2022-08-18 | Edigene Therapeutics (Beijing) Inc. | New method for targeted editing of RNA |
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 |
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2020
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CN114269919A (zh) | 2022-04-01 |
ECSP22010372A (es) | 2022-03-31 |
CL2022000080A1 (es) | 2022-11-18 |
CR20220063A (es) | 2022-07-22 |
AU2020313143B2 (en) | 2024-04-04 |
KR20220038706A (ko) | 2022-03-29 |
CA3146771A1 (en) | 2021-01-21 |
JP2022536546A (ja) | 2022-08-17 |
CN117778385A (zh) | 2024-03-29 |
BR112022000291A2 (pt) | 2022-03-15 |
CO2022001359A2 (es) | 2022-05-10 |
WO2021008447A1 (en) | 2021-01-21 |
US11661596B2 (en) | 2023-05-30 |
CN114269919B (zh) | 2023-08-18 |
AU2020313143A1 (en) | 2022-02-10 |
EP3997229A1 (en) | 2022-05-18 |
US20230365962A1 (en) | 2023-11-16 |
PE20220300A1 (es) | 2022-03-09 |
IL289700A (en) | 2022-03-01 |
US20220135963A1 (en) | 2022-05-05 |
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