BR112018074930A2 - ácido nucleico, vetor de ácido nucleico, partícula de entrega, composição farmacêutica, célula hospedeira, método para edição do genoma e método e kit para prevenção e/ou tratamento de uma doença - Google Patents
ácido nucleico, vetor de ácido nucleico, partícula de entrega, composição farmacêutica, célula hospedeira, método para edição do genoma e método e kit para prevenção e/ou tratamento de uma doençaInfo
- Publication number
- BR112018074930A2 BR112018074930A2 BR112018074930-3A BR112018074930A BR112018074930A2 BR 112018074930 A2 BR112018074930 A2 BR 112018074930A2 BR 112018074930 A BR112018074930 A BR 112018074930A BR 112018074930 A2 BR112018074930 A2 BR 112018074930A2
- Authority
- BR
- Brazil
- Prior art keywords
- nucleic acid
- kit
- treatment
- pharmaceutical composition
- host cell
- Prior art date
Links
- 108020004707 nucleic acids Proteins 0.000 title abstract 7
- 102000039446 nucleic acids Human genes 0.000 title abstract 7
- 150000007523 nucleic acids Chemical class 0.000 title abstract 7
- 238000000034 method Methods 0.000 title 2
- 230000006806 disease prevention Effects 0.000 title 1
- 238000010362 genome editing Methods 0.000 title 1
- 239000002245 particle Substances 0.000 title 1
- 239000008194 pharmaceutical composition Substances 0.000 title 1
- 101710163270 Nuclease Proteins 0.000 abstract 4
- RXWNCPJZOCPEPQ-NVWDDTSBSA-N puromycin Chemical compound C1=CC(OC)=CC=C1C[C@H](N)C(=O)N[C@H]1[C@@H](O)[C@H](N2C3=NC=NC(=C3N=C2)N(C)C)O[C@@H]1CO RXWNCPJZOCPEPQ-NVWDDTSBSA-N 0.000 abstract 4
- 230000001105 regulatory effect Effects 0.000 abstract 3
- 230000004927 fusion Effects 0.000 abstract 2
- 108091033319 polynucleotide Proteins 0.000 abstract 2
- 102000040430 polynucleotide Human genes 0.000 abstract 2
- 239000002157 polynucleotide Substances 0.000 abstract 2
- 108090000623 proteins and genes Proteins 0.000 abstract 2
- 229950010131 puromycin Drugs 0.000 abstract 2
- 108091027981 Response element Proteins 0.000 abstract 1
- 210000001744 T-lymphocyte Anatomy 0.000 abstract 1
- YJQCOFNZVFGCAF-UHFFFAOYSA-N Tunicamycin II Natural products O1C(CC(O)C2C(C(O)C(O2)N2C(NC(=O)C=C2)=O)O)C(O)C(O)C(NC(=O)C=CCCCCCCCCC(C)C)C1OC1OC(CO)C(O)C(O)C1NC(C)=O YJQCOFNZVFGCAF-UHFFFAOYSA-N 0.000 abstract 1
- 150000001413 amino acids Chemical class 0.000 abstract 1
- 210000004027 cell Anatomy 0.000 abstract 1
- 230000002950 deficient Effects 0.000 abstract 1
- 230000037213 diet Effects 0.000 abstract 1
- 235000005911 diet Nutrition 0.000 abstract 1
- 235000020776 essential amino acid Nutrition 0.000 abstract 1
- 239000003797 essential amino acid Substances 0.000 abstract 1
- 230000010354 integration Effects 0.000 abstract 1
- 239000013612 plasmid Substances 0.000 abstract 1
- ZHSGGJXRNHWHRS-VIDYELAYSA-N tunicamycin Chemical compound O([C@H]1[C@@H]([C@H]([C@@H](O)[C@@H](CC(O)[C@@H]2[C@H]([C@@H](O)[C@@H](O2)N2C(NC(=O)C=C2)=O)O)O1)O)NC(=O)/C=C/CC(C)C)[C@H]1O[C@H](CO)[C@@H](O)[C@H](O)[C@H]1NC(C)=O ZHSGGJXRNHWHRS-VIDYELAYSA-N 0.000 abstract 1
- MEYZYGMYMLNUHJ-UHFFFAOYSA-N tunicamycin Natural products CC(C)CCCCCCCCCC=CC(=O)NC1C(O)C(O)C(CC(O)C2OC(C(O)C2O)N3C=CC(=O)NC3=O)OC1OC4OC(CO)C(O)C(O)C4NC(=O)C MEYZYGMYMLNUHJ-UHFFFAOYSA-N 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
- 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
-
- 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]
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
-
- 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
- A61P35/00—Antineoplastic agents
- A61P35/02—Antineoplastic agents specific for leukemia
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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]
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
- C12N2830/002—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Oncology (AREA)
- Epidemiology (AREA)
- Mycology (AREA)
- Hematology (AREA)
- Communicable Diseases (AREA)
- Cell Biology (AREA)
- Crystallography & Structural Chemistry (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Enzymes And Modification Thereof (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP16172964 | 2016-06-03 | ||
| EPEP16172964 | 2016-06-03 | ||
| PCT/EP2017/063549 WO2017207797A1 (en) | 2016-06-03 | 2017-06-02 | Diet controlled expression of a nucleic acid encoding cas9 nuclease and uses thereof |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112018074930A2 true BR112018074930A2 (pt) | 2019-03-12 |
Family
ID=56148119
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112018074930-3A BR112018074930A2 (pt) | 2016-06-03 | 2017-06-02 | ácido nucleico, vetor de ácido nucleico, partícula de entrega, composição farmacêutica, célula hospedeira, método para edição do genoma e método e kit para prevenção e/ou tratamento de uma doença |
Country Status (11)
| Country | Link |
|---|---|
| US (2) | US20190185832A1 (enExample) |
| JP (2) | JP7436145B2 (enExample) |
| KR (1) | KR102317622B1 (enExample) |
| CN (2) | CN109906271A (enExample) |
| AU (1) | AU2017275769B2 (enExample) |
| BR (1) | BR112018074930A2 (enExample) |
| CA (1) | CA3025591A1 (enExample) |
| IL (1) | IL263291B2 (enExample) |
| RU (1) | RU2771383C2 (enExample) |
| SG (1) | SG11201810772XA (enExample) |
| WO (1) | WO2017207797A1 (enExample) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3823995A4 (en) * | 2018-08-01 | 2022-05-04 | The Regents of the University of Colorado, a body corporate | PROGRAMMABLE THERAPEUTIC FUSOGENE SECRETED GECTOSOME DESIGNER VESICLES FOR MACROMOLECULE RELEASE AND GENOME MODIFICATION |
| CN115820654A (zh) * | 2019-08-30 | 2023-03-21 | 深圳华大基因股份有限公司 | Loxhd1基因突变体及其应用 |
| CN114058689B (zh) * | 2020-07-30 | 2024-08-20 | 南京市妇幼保健院 | 一种基因突变检测试剂盒及其应用 |
| CN119894540A (zh) * | 2022-07-05 | 2025-04-25 | 营养治疗基因公司 | 人类t或nk细胞中转基因的受控表达用于细胞免疫疗法 |
| WO2024100176A1 (en) * | 2022-11-10 | 2024-05-16 | Nutritheragene | Controlled gene therapy of ocular diseases |
| CN116732043B (zh) * | 2023-08-10 | 2023-10-17 | 四川省医学科学院·四川省人民医院 | 一种突变基因及其白内障筛查的用途与白内障筛查试剂盒 |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| FR2982277B1 (fr) | 2011-11-08 | 2015-08-21 | Agronomique Inst Nat Rech | Cassette d'expression inductible et ses utilisations |
| KR102479178B1 (ko) * | 2012-12-06 | 2022-12-19 | 시그마-알드리치 컴퍼니., 엘엘씨 | Crispr-기초된 유전체 변형과 조절 |
| US20150098954A1 (en) * | 2013-10-08 | 2015-04-09 | Elwha Llc | Compositions and Methods Related to CRISPR Targeting |
| WO2015089462A1 (en) * | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing |
| SG10201804975PA (en) * | 2013-12-12 | 2018-07-30 | Broad Inst Inc | Delivery, Use and Therapeutic Applications of the Crispr-Cas Systems and Compositions for HBV and Viral Diseases and Disorders |
-
2017
- 2017-06-02 SG SG11201810772XA patent/SG11201810772XA/en unknown
- 2017-06-02 RU RU2018142174A patent/RU2771383C2/ru active
- 2017-06-02 CN CN201780045274.4A patent/CN109906271A/zh active Pending
- 2017-06-02 AU AU2017275769A patent/AU2017275769B2/en active Active
- 2017-06-02 BR BR112018074930-3A patent/BR112018074930A2/pt not_active Application Discontinuation
- 2017-06-02 CA CA3025591A patent/CA3025591A1/en active Pending
- 2017-06-02 CN CN202211117939.7A patent/CN116064534A/zh active Pending
- 2017-06-02 WO PCT/EP2017/063549 patent/WO2017207797A1/en not_active Ceased
- 2017-06-02 KR KR1020197000200A patent/KR102317622B1/ko active Active
- 2017-06-02 JP JP2018563152A patent/JP7436145B2/ja active Active
- 2017-06-02 US US16/304,988 patent/US20190185832A1/en not_active Abandoned
-
2018
- 2018-11-26 IL IL263291A patent/IL263291B2/en unknown
-
2022
- 2022-07-07 JP JP2022110001A patent/JP2022133441A/ja active Pending
-
2023
- 2023-01-12 US US18/153,490 patent/US20230313161A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| CN109906271A (zh) | 2019-06-18 |
| KR20190031230A (ko) | 2019-03-25 |
| RU2771383C2 (ru) | 2022-05-04 |
| US20230313161A1 (en) | 2023-10-05 |
| CA3025591A1 (en) | 2017-12-07 |
| WO2017207797A1 (en) | 2017-12-07 |
| AU2017275769A1 (en) | 2018-12-20 |
| SG11201810772XA (en) | 2018-12-28 |
| IL263291B2 (en) | 2023-07-01 |
| AU2017275769B2 (en) | 2023-04-13 |
| RU2018142174A3 (enExample) | 2020-09-30 |
| JP7436145B2 (ja) | 2024-02-21 |
| US20190185832A1 (en) | 2019-06-20 |
| IL263291A (en) | 2018-12-31 |
| CN116064534A (zh) | 2023-05-05 |
| JP2022133441A (ja) | 2022-09-13 |
| KR102317622B1 (ko) | 2021-10-26 |
| RU2018142174A (ru) | 2020-07-10 |
| IL263291B1 (en) | 2023-03-01 |
| JP2019517262A (ja) | 2019-06-24 |
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| B350 | Update of information on the portal [chapter 15.35 patent gazette] | ||
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| B07A | Application suspended after technical examination (opinion) [chapter 7.1 patent gazette] | ||
| B09B | Patent application refused [chapter 9.2 patent gazette] | ||
| B12B | Appeal against refusal [chapter 12.2 patent gazette] |