BR112021007403A2 - Composições e métodos para entrega de transgenes - Google Patents
Composições e métodos para entrega de transgenesInfo
- Publication number
- BR112021007403A2 BR112021007403A2 BR112021007403A BR112021007403A BR112021007403A2 BR 112021007403 A2 BR112021007403 A2 BR 112021007403A2 BR 112021007403 A BR112021007403 A BR 112021007403A BR 112021007403 A BR112021007403 A BR 112021007403A BR 112021007403 A2 BR112021007403 A2 BR 112021007403A2
- Authority
- BR
- Brazil
- Prior art keywords
- compositions
- methods
- systems
- transgene
- transgene delivery
- Prior art date
Links
- 238000000034 method Methods 0.000 title abstract 6
- 239000000203 mixture Substances 0.000 title abstract 6
- 108700019146 Transgenes Proteins 0.000 title abstract 3
- 238000010362 genome editing Methods 0.000 abstract 2
- 108010088751 Albumins Proteins 0.000 abstract 1
- -1 DNA and RNA Chemical class 0.000 abstract 1
- 238000001727 in vivo Methods 0.000 abstract 1
- 108020004707 nucleic acids Proteins 0.000 abstract 1
- 150000007523 nucleic acids Chemical class 0.000 abstract 1
- 102000039446 nucleic acids Human genes 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 230000008685 targeting Effects 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
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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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- 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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/745—Blood coagulation or fibrinolysis factors
- C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/81—Protease inhibitors
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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
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- 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 RNAses, DNAses
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
- C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
- C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
- C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
- C12N9/6424—Serine endopeptidases (3.4.21)
- C12N9/644—Coagulation factor IXa (3.4.21.22)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/21—Serine endopeptidases (3.4.21)
- C12Y304/21022—Coagulation factor IXa (3.4.21.22)
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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
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- 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 [CRISPRs]
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- C—CHEMISTRY; METALLURGY
- 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
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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
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Pharmacology & Pharmacy (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Hematology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Cell Biology (AREA)
- Mycology (AREA)
- Epidemiology (AREA)
- Toxicology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Diabetes (AREA)
- Crystallography & Structural Chemistry (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicinal Preparation (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Abstract
composições e métodos para entrega de transgenes. trata-se de composições, métodos e sistemas para distribuição direcionada de ácidos nucleicos, incluindo dna e rna, para uma célula alvo. também são fornecidos composições, métodos e sistemas para expressar um transgene em uma célula por edição genômica. além disso, são fornecidas composições, métodos e sistemas para bater em um gene de interesse (goi) em um locus genômico alvo no genoma, em particular o locus do gene da albumina. também são fornecidos composições, métodos e sistemas para o tratamento de um sujeito com ou suspeito de ter um distúrbio ou condição de saúde empregando edição do genoma ex vivo e / ou in vivo.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201862747128P | 2018-10-17 | 2018-10-17 | |
PCT/US2019/056779 WO2020081843A1 (en) | 2018-10-17 | 2019-10-17 | Compositions and methods for delivering transgenes |
Publications (1)
Publication Number | Publication Date |
---|---|
BR112021007403A2 true BR112021007403A2 (pt) | 2021-12-07 |
Family
ID=68468827
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112021007403A BR112021007403A2 (pt) | 2018-10-17 | 2019-10-17 | Composições e métodos para entrega de transgenes |
Country Status (11)
Country | Link |
---|---|
US (1) | US20210348159A1 (pt) |
EP (1) | EP3867377A1 (pt) |
JP (1) | JP2022505173A (pt) |
KR (1) | KR20210096088A (pt) |
CN (1) | CN113366106A (pt) |
AU (1) | AU2019362000A1 (pt) |
BR (1) | BR112021007403A2 (pt) |
CA (1) | CA3116885A1 (pt) |
IL (1) | IL282369A (pt) |
MX (1) | MX2021004455A (pt) |
WO (1) | WO2020081843A1 (pt) |
Families Citing this family (5)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
MX2021015122A (es) | 2019-06-07 | 2022-04-06 | Regeneron Pharma | Animales no humanos que comprenden un locus de albumina humanizado. |
MX2023004811A (es) * | 2020-10-26 | 2023-06-29 | Res Institute At Nationwide Children´S Hospital | Células nk del receptor de antígeno quimérico (car) y usos de las mismas. |
CN114496063B (zh) * | 2022-01-13 | 2023-05-23 | 北京博康健基因科技有限公司 | 基于天然氨基酸序列的信号肽设计与二级结构从头算建模方法和装置 |
WO2024006960A1 (en) * | 2022-06-29 | 2024-01-04 | Juno Therapeutics, Inc. | Lipid nanoparticles for delivery of nucleic acids |
CN117947040A (zh) * | 2022-10-31 | 2024-04-30 | 苏州荷光科汇生物科技有限公司 | 用于目的基因的表达盒及其应用 |
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-
2019
- 2019-10-17 AU AU2019362000A patent/AU2019362000A1/en active Pending
- 2019-10-17 EP EP19798448.7A patent/EP3867377A1/en active Pending
- 2019-10-17 KR KR1020217014866A patent/KR20210096088A/ko active Search and Examination
- 2019-10-17 BR BR112021007403A patent/BR112021007403A2/pt unknown
- 2019-10-17 CA CA3116885A patent/CA3116885A1/en active Pending
- 2019-10-17 JP JP2021521116A patent/JP2022505173A/ja active Pending
- 2019-10-17 US US17/286,276 patent/US20210348159A1/en active Pending
- 2019-10-17 CN CN201980083482.2A patent/CN113366106A/zh active Pending
- 2019-10-17 MX MX2021004455A patent/MX2021004455A/es unknown
- 2019-10-17 WO PCT/US2019/056779 patent/WO2020081843A1/en unknown
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MX2021004455A (es) | 2021-08-11 |
WO2020081843A1 (en) | 2020-04-23 |
IL282369A (en) | 2021-06-30 |
US20210348159A1 (en) | 2021-11-11 |
EP3867377A1 (en) | 2021-08-25 |
AU2019362000A1 (en) | 2021-05-20 |
CA3116885A1 (en) | 2020-04-23 |
CN113366106A (zh) | 2021-09-07 |
KR20210096088A (ko) | 2021-08-04 |
JP2022505173A (ja) | 2022-01-14 |
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