TW202320864A - 經工程改造之casx抑制子系統 - Google Patents
經工程改造之casx抑制子系統Info
- Publication number
- TW202320864A TW202320864A TW111135799A TW111135799A TW202320864A TW 202320864 A TW202320864 A TW 202320864A TW 111135799 A TW111135799 A TW 111135799A TW 111135799 A TW111135799 A TW 111135799A TW 202320864 A TW202320864 A TW 202320864A
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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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- 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
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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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
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing 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
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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
- 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
- C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
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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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/10—Transferases (2.)
- C12N9/1003—Transferases (2.) transferring one-carbon groups (2.1)
- C12N9/1007—Methyltransferases (general) (2.1.1.)
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
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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
- 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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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Virology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Mycology (AREA)
- Cell Biology (AREA)
- Crystallography & Structural Chemistry (AREA)
- Peptides Or Proteins (AREA)
- Apparatus Associated With Microorganisms And Enzymes (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (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 |
|---|---|---|---|
| US202163246543P | 2021-09-21 | 2021-09-21 | |
| US63/246,543 | 2021-09-21 | ||
| US202263321517P | 2022-03-18 | 2022-03-18 | |
| US63/321,517 | 2022-03-18 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| TW202320864A true TW202320864A (zh) | 2023-06-01 |
Family
ID=83902730
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| TW111135799A TW202320864A (zh) | 2021-09-21 | 2022-09-21 | 經工程改造之casx抑制子系統 |
Country Status (13)
| Country | Link |
|---|---|
| US (3) | US20240392271A1 (enExample) |
| EP (1) | EP4405479A2 (enExample) |
| JP (1) | JP2024534523A (enExample) |
| KR (1) | KR20240095525A (enExample) |
| AU (1) | AU2022349627A1 (enExample) |
| CA (1) | CA3231909A1 (enExample) |
| CL (1) | CL2024000835A1 (enExample) |
| GB (1) | GB2625500B (enExample) |
| IL (1) | IL311610A (enExample) |
| MX (1) | MX2024003455A (enExample) |
| PE (1) | PE20240728A1 (enExample) |
| TW (1) | TW202320864A (enExample) |
| WO (1) | WO2023049742A2 (enExample) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN117143257A (zh) * | 2023-10-31 | 2023-12-01 | 深圳市帝迈生物技术有限公司 | Trim28-krab-znf10二元复合物、制备方法及用于前列腺癌筛查的试剂盒 |
Families Citing this family (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG11202113253SA (en) | 2019-06-07 | 2021-12-30 | Scribe Therapeutics Inc | Engineered casx systems |
| AU2021392719B2 (en) | 2020-12-03 | 2025-08-14 | Scribe Therapeutics Inc. | Engineered class 2 type v crispr systems |
| WO2022261150A2 (en) | 2021-06-09 | 2022-12-15 | Scribe Therapeutics Inc. | Particle delivery systems |
| IL311610A (en) | 2021-09-21 | 2024-05-01 | Scribe Therapeutics Inc | Engineered CASX suppressor systems |
| US20230190961A1 (en) | 2021-10-20 | 2023-06-22 | University Of Rochester | Compositions and methods for treating myelin deficiency by rejuvenating glial progenitor cells |
| WO2023173110A1 (en) * | 2022-03-11 | 2023-09-14 | Epicrispr Biotechnologies, Inc. | Compositions, systems, and methods for treating familial hypercholesterolemia by targeting pcsk9 |
| WO2023235726A2 (en) * | 2022-05-31 | 2023-12-07 | Regeneron Pharmaceuticals, Inc. | Crispr interference therapeutics for c9orf72 repeat expansion disease |
| CA3257824A1 (en) * | 2022-06-02 | 2023-12-07 | Scribe Therapeutics Inc. | MODIFIED TYPE V, CLASS 2 CRISPR SYSTEMS |
| WO2023235888A2 (en) | 2022-06-03 | 2023-12-07 | Scribe Therapeutics Inc. | COMPOSITIONS AND METHODS FOR CpG DEPLETION |
| EP4536819A1 (en) | 2022-06-07 | 2025-04-16 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of pcsk9 |
| WO2023240027A1 (en) | 2022-06-07 | 2023-12-14 | Scribe Therapeutics Inc. | Particle delivery systems |
| CN119998446A (zh) * | 2022-06-07 | 2025-05-13 | 斯克里贝治疗公司 | 用于靶向pcsk9的组合物和方法 |
| EP4536286A1 (en) | 2022-06-08 | 2025-04-16 | Scribe Therapeutics Inc. | Aav vectors for gene editing |
| CA3260474A1 (en) * | 2022-06-23 | 2023-12-28 | Chroma Medicine, Inc. | COMPOSITIONS AND METHODS OF EPIGENETIC REGULATION OF B2M EXPRESSION |
| CA3260237A1 (en) | 2022-06-24 | 2023-12-28 | Tune Therapeutics, Inc. | Compositions, systems and methods for reducing low-density lipoprotein by targeted gene repression |
| WO2024206555A1 (en) | 2023-03-29 | 2024-10-03 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of pcsk9 |
| TW202444914A (zh) | 2023-03-29 | 2024-11-16 | 美商斯奎柏治療公司 | 抑制子融合蛋白系統 |
| WO2024223911A1 (en) * | 2023-04-28 | 2024-10-31 | Ziccum Ab | Nanoparticle formulations |
| WO2024229020A2 (en) * | 2023-05-01 | 2024-11-07 | Chroma Medicine, Inc. | Compositions and methods for epigenetic regulation of pcsk9 expression |
| WO2025072293A1 (en) * | 2023-09-27 | 2025-04-03 | Scribe Therapeutics Inc. | Optimized mrnas encoding casx proteins |
| WO2025101979A1 (en) * | 2023-11-08 | 2025-05-15 | Nchroma Bio, Inc. | Compositions and methods for epigenetic regulation of genes for treatment of non-alcoholic steatohepatitis or metabolic dysfunction-associated steatohepatitis |
| CN117887718B (zh) * | 2024-03-14 | 2025-02-28 | 青岛宝迈得生物科技有限公司 | 一种mettl6基因抑制剂及其应用 |
| CN120966794A (zh) * | 2024-05-16 | 2025-11-18 | 君福生物科技(广州)有限公司 | 用于表观遗传编辑的融合蛋白及其应用 |
| CN118931878B (zh) * | 2024-10-14 | 2025-02-07 | 内蒙古大学 | 一种基于CRISPR/dCas9的表观遗传基因编辑系统及其应用 |
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Cited By (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN117143257A (zh) * | 2023-10-31 | 2023-12-01 | 深圳市帝迈生物技术有限公司 | Trim28-krab-znf10二元复合物、制备方法及用于前列腺癌筛查的试剂盒 |
| CN117143257B (zh) * | 2023-10-31 | 2024-02-09 | 深圳市帝迈生物技术有限公司 | Trim28-krab-znf10二元复合物、制备方法及用于前列腺癌筛查的试剂盒 |
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| CL2024000835A1 (es) | 2024-10-04 |
| WO2023049742A2 (en) | 2023-03-30 |
| US20240254466A1 (en) | 2024-08-01 |
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