PE20220256A1 - Sistemas de casx artificiales - Google Patents
Sistemas de casx artificialesInfo
- Publication number
- PE20220256A1 PE20220256A1 PE2021002050A PE2021002050A PE20220256A1 PE 20220256 A1 PE20220256 A1 PE 20220256A1 PE 2021002050 A PE2021002050 A PE 2021002050A PE 2021002050 A PE2021002050 A PE 2021002050A PE 20220256 A1 PE20220256 A1 PE 20220256A1
- Authority
- PE
- Peru
- Prior art keywords
- casx
- variant
- artificial
- systems
- nag
- Prior art date
Links
- 102000004169 proteins and genes Human genes 0.000 abstract 3
- 108090000623 proteins and genes Proteins 0.000 abstract 3
- 102000004389 Ribonucleoproteins Human genes 0.000 abstract 1
- 108010081734 Ribonucleoproteins Proteins 0.000 abstract 1
- 102000039446 nucleic acids Human genes 0.000 abstract 1
- 108020004707 nucleic acids Proteins 0.000 abstract 1
- 150000007523 nucleic acids Chemical class 0.000 abstract 1
Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/10—Applications; Uses in screening processes
- C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2800/00—Nucleic acids vectors
- C12N2800/10—Plasmid DNA
- C12N2800/106—Plasmid DNA for vertebrates
- C12N2800/107—Plasmid DNA for vertebrates for mammalian
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Medicinal Chemistry (AREA)
- Virology (AREA)
- Cell Biology (AREA)
- Mycology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962858750P | 2019-06-07 | 2019-06-07 | |
| US201962944892P | 2019-12-06 | 2019-12-06 | |
| US202063030838P | 2020-05-27 | 2020-05-27 | |
| PCT/US2020/036505 WO2020247882A1 (en) | 2019-06-07 | 2020-06-05 | Engineered casx systems |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PE20220256A1 true PE20220256A1 (es) | 2022-02-21 |
Family
ID=71899867
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PE2021002050A PE20220256A1 (es) | 2019-06-07 | 2020-06-05 | Sistemas de casx artificiales |
Country Status (17)
| Country | Link |
|---|---|
| US (5) | US11560555B2 (enExample) |
| EP (1) | EP3980533A1 (enExample) |
| JP (2) | JP7744011B2 (enExample) |
| KR (1) | KR20220032050A (enExample) |
| CN (1) | CN114375334B (enExample) |
| AU (1) | AU2020289591A1 (enExample) |
| BR (1) | BR112021024288A2 (enExample) |
| CA (1) | CA3142883A1 (enExample) |
| CL (1) | CL2021003233A1 (enExample) |
| GB (1) | GB2600274B (enExample) |
| IL (1) | IL288738A (enExample) |
| MX (1) | MX2021015058A (enExample) |
| PE (1) | PE20220256A1 (enExample) |
| PH (1) | PH12021553076A1 (enExample) |
| SG (1) | SG11202113253SA (enExample) |
| TW (1) | TW202113074A (enExample) |
| WO (1) | WO2020247882A1 (enExample) |
Families Citing this family (45)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12275964B2 (en) | 2018-08-22 | 2025-04-15 | The Regents Of The University Of California | Variant type V CRISPR/Cas effector polypeptides and methods of use thereof |
| EP3980533A1 (en) | 2019-06-07 | 2022-04-13 | Scribe Therapeutics Inc. | Engineered casx systems |
| WO2021050601A1 (en) | 2019-09-09 | 2021-03-18 | Scribe Therapeutics Inc. | Compositions and methods for use in immunotherapy |
| WO2021050593A1 (en) | 2019-09-09 | 2021-03-18 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of sod1 |
| WO2021113763A1 (en) | 2019-12-06 | 2021-06-10 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of rhodopsin |
| IL294620A (en) * | 2020-01-10 | 2022-09-01 | Scribe Therapeutics Inc | Compositions and methods for the targeting of pcsk9 |
| AU2021237633A1 (en) | 2020-03-18 | 2022-10-06 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of C9orf72 |
| GB202010075D0 (en) * | 2020-07-01 | 2020-08-12 | Imp College Innovations Ltd | Therapeutic nucleic acids, peptides and uses |
| WO2022119957A1 (en) * | 2020-12-02 | 2022-06-09 | The Regents Of The University Of California | Crispr-cas effector polypeptides and methods of use thereof |
| KR20230128289A (ko) | 2020-12-03 | 2023-09-04 | 스크라이브 테라퓨틱스 인크. | 조작된 클래스 2 유형 v crispr 시스템 |
| US20240100185A1 (en) | 2020-12-03 | 2024-03-28 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of ptbp1 |
| WO2022125843A1 (en) | 2020-12-09 | 2022-06-16 | Scribe Therapeutics Inc. | Aav vectors for gene editing |
| TW202302844A (zh) * | 2021-03-09 | 2023-01-16 | 美商阿伯生物技術公司 | 包含變異多肽之組合物及其用途 |
| EP4347818A2 (en) | 2021-06-01 | 2024-04-10 | Arbor Biotechnologies, Inc. | Gene editing systems comprising a crispr nuclease and uses thereof |
| WO2022261149A2 (en) | 2021-06-09 | 2022-12-15 | Scribe Therapeutics Inc. | Particle delivery systems |
| GB2625500B (en) | 2021-09-21 | 2026-04-01 | Scribe Therapeutics Inc | Engineered CasX repressor systems |
| EP4405490A2 (en) | 2021-09-23 | 2024-07-31 | Scribe Therapeutics Inc. | Self-inactivating vectors for gene editing |
| CN116497002A (zh) * | 2022-01-19 | 2023-07-28 | 中国科学院动物研究所 | 工程化的CasX核酸酶、效应蛋白及其用途 |
| WO2023235818A2 (en) | 2022-06-02 | 2023-12-07 | Scribe Therapeutics Inc. | Engineered class 2 type v crispr systems |
| WO2023235888A2 (en) | 2022-06-03 | 2023-12-07 | Scribe Therapeutics Inc. | COMPOSITIONS AND METHODS FOR CpG DEPLETION |
| TW202413643A (zh) | 2022-06-07 | 2024-04-01 | 美商斯奎柏治療公司 | 用於靶向pcsk9的組合物及方法 |
| WO2023240027A1 (en) | 2022-06-07 | 2023-12-14 | Scribe Therapeutics Inc. | Particle delivery systems |
| GB2634837A (en) | 2022-06-07 | 2025-04-23 | Scribe Therapeutics Inc | Compositions and methods for the targeting of PCSK9 |
| US20250361525A1 (en) | 2022-06-08 | 2025-11-27 | Scribe Therapeutics Inc. | Aav vectors for gene editing |
| WO2023240157A2 (en) | 2022-06-08 | 2023-12-14 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of dmd |
| WO2024081711A2 (en) * | 2022-10-11 | 2024-04-18 | The Broad Institute, Inc. | Reprogramable tnpb polypeptides and use thereof |
| KR20250151430A (ko) | 2023-02-15 | 2025-10-21 | 아버 바이오테크놀로지스, 인크. | 스타스민 2(stmn2) 전사체에서 비정상적인 스플라이싱을 억제하는 유전자 편집 방법 |
| AU2024236558A1 (en) | 2023-03-15 | 2025-10-09 | Renagade Therapeutics Management Inc. | Delivery of gene editing systems and methods of use thereof |
| WO2024206565A1 (en) | 2023-03-29 | 2024-10-03 | Scribe Therapeutics Inc. | Repressor fusion protein systems |
| PE20252746A1 (es) | 2023-03-29 | 2025-12-05 | Scribe Therapeutics Inc | Composiciones y metodos para el direccionamiento a pcsk9 |
| WO2024206620A1 (en) | 2023-03-29 | 2024-10-03 | Scribe Therapeutics Inc. | Messenger rna encoding casx |
| WO2024206676A1 (en) | 2023-03-29 | 2024-10-03 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of lpa |
| CN116926043B (zh) * | 2023-04-28 | 2024-09-06 | 新乡医学院第三附属医院 | 一种dCasX蛋白核酸酶、重组载体及其应用 |
| EP4705445A2 (en) * | 2023-05-02 | 2026-03-11 | Inari Agriculture Technology, Inc. | Ribozyme sequences for processing rna |
| AU2024270764A1 (en) | 2023-05-15 | 2025-12-04 | Nchroma Bio, Inc. | Compositions and methods for epigenetic regulation of hbv gene expression |
| JPWO2024257843A1 (enExample) * | 2023-06-16 | 2024-12-19 | ||
| AU2024335327A1 (en) | 2023-09-01 | 2026-03-26 | Renagade Therapeutics Management Inc. | Gene editing systems, compositions, and methods for treatment of vexas syndrome |
| WO2025072293A1 (en) | 2023-09-27 | 2025-04-03 | Scribe Therapeutics Inc. | Optimized mrnas encoding casx proteins |
| CN117296799B (zh) * | 2023-11-28 | 2024-02-02 | 四川省医学科学院·四川省人民医院 | 一种视网膜色素变性疾病模型的构建方法及其应用 |
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2020
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| US11560555B2 (en) | 2023-01-24 |
| US20230124880A1 (en) | 2023-04-20 |
| WO2020247882A1 (en) | 2020-12-10 |
| US20250136962A1 (en) | 2025-05-01 |
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| JP7744011B2 (ja) | 2025-09-25 |
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| US20220220508A1 (en) | 2022-07-14 |
| US20220081681A1 (en) | 2022-03-17 |
| KR20220032050A (ko) | 2022-03-15 |
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| CA3142883A1 (en) | 2020-12-10 |
| CL2021003233A1 (es) | 2022-08-12 |
| TW202113074A (zh) | 2021-04-01 |
| JP2022534809A (ja) | 2022-08-03 |
| IL288738A (en) | 2022-02-01 |
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| CN114375334B (zh) | 2026-03-20 |
| GB2600274A (en) | 2022-04-27 |
| US12084692B2 (en) | 2024-09-10 |
| EP3980533A1 (en) | 2022-04-13 |
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| US20250043262A1 (en) | 2025-02-06 |
| BR112021024288A2 (pt) | 2022-02-15 |
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