BR112022008861A2 - Construto de ácido nucleico, vetor, nanopartícula lipídica, célula, composição para uso na expressão de retinosquisina, método para integrar uma sequência de codificação, e, método para modificar um gene rs1 - Google Patents
Construto de ácido nucleico, vetor, nanopartícula lipídica, célula, composição para uso na expressão de retinosquisina, método para integrar uma sequência de codificação, e, método para modificar um gene rs1Info
- Publication number
- BR112022008861A2 BR112022008861A2 BR112022008861A BR112022008861A BR112022008861A2 BR 112022008861 A2 BR112022008861 A2 BR 112022008861A2 BR 112022008861 A BR112022008861 A BR 112022008861A BR 112022008861 A BR112022008861 A BR 112022008861A BR 112022008861 A2 BR112022008861 A2 BR 112022008861A2
- Authority
- BR
- Brazil
- Prior art keywords
- nucleic acid
- retinoschisin
- cell
- coding sequence
- integrating
- Prior art date
Links
- 238000000034 method Methods 0.000 title abstract 6
- 239000000203 mixture Substances 0.000 title abstract 5
- 108020004707 nucleic acids Proteins 0.000 title abstract 4
- 150000007523 nucleic acids Chemical class 0.000 title abstract 4
- 102000039446 nucleic acids Human genes 0.000 title abstract 4
- 108091026890 Coding region Proteins 0.000 title abstract 3
- 101710111169 Retinoschisin Proteins 0.000 title abstract 3
- 102100039507 Retinoschisin Human genes 0.000 title abstract 3
- -1 CELL Substances 0.000 title abstract 2
- 238000010276 construction Methods 0.000 title abstract 2
- 150000002632 lipids Chemical class 0.000 title abstract 2
- 239000002105 nanoparticle Substances 0.000 title abstract 2
- 108090000623 proteins and genes Proteins 0.000 title abstract 2
- 101710163270 Nuclease Proteins 0.000 abstract 1
- 208000017441 X-linked retinoschisis Diseases 0.000 abstract 1
- 239000003795 chemical substances by application Substances 0.000 abstract 1
- 238000003780 insertion Methods 0.000 abstract 1
- 230000037431 insertion Effects 0.000 abstract 1
- 230000010354 integration Effects 0.000 abstract 1
- 201000007714 retinoschisis Diseases 0.000 abstract 1
- 230000008685 targeting Effects 0.000 abstract 1
Classifications
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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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K67/00—Rearing or breeding animals, not otherwise provided for; New breeds of animals
- A01K67/027—New breeds of vertebrates
- A01K67/0275—Genetically modified vertebrates, e.g. transgenic
- A01K67/0278—Humanized animals, e.g. knockin
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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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- 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
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic 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
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
- C07K14/7056—Lectin superfamily, e.g. CD23, CD72
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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
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing nucleic acids
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- 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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- 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
- 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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- 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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- 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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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
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- 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 [CRISPRs]
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- C—CHEMISTRY; METALLURGY
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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
Abstract
CONSTRUTO DE ÁCIDO NUCLEICO, VETOR, NANOPARTÍCULA LIPÍDICA, CÉLULA, COMPOSIÇÃO PARA USO NA EXPRESSÃO DE RETINOSQUISINA, MÉTODO PARA INTEGRAR UMA SEQUÊNCIA DE CODIFICAÇÃO, E, MÉTODO PARA MODIFICAR UM GENE RS1. Trata se de construtos e composições de ácido nucleico que permitem a inserção e/ou expressão de uma sequência de codificação de retinosquisina. São fornecidos agentes de nuclease direcionados aos loci de RS1. São fornecidos, também, composições e métodos de uso de tais construtos para integração em um locus alvo genômico e/ou expressão em uma célula. São fornecidos, também, métodos de tratamento de retinosquise juvenil ligada ao X que usam os construtos e composições de ácido nucleico.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201962932608P | 2019-11-08 | 2019-11-08 | |
PCT/US2020/059568 WO2021092513A1 (en) | 2019-11-08 | 2020-11-07 | Crispr and aav strategies for x-linked juvenile retinoschisis therapy |
Publications (1)
Publication Number | Publication Date |
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BR112022008861A2 true BR112022008861A2 (pt) | 2022-08-23 |
Family
ID=73654921
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
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BR112022008861A BR112022008861A2 (pt) | 2019-11-08 | 2020-11-07 | Construto de ácido nucleico, vetor, nanopartícula lipídica, célula, composição para uso na expressão de retinosquisina, método para integrar uma sequência de codificação, e, método para modificar um gene rs1 |
Country Status (11)
Country | Link |
---|---|
US (1) | US20230001019A1 (pt) |
EP (1) | EP4054651A1 (pt) |
JP (1) | JP2022553573A (pt) |
KR (1) | KR20220097414A (pt) |
CN (1) | CN114746125A (pt) |
AU (1) | AU2020379046A1 (pt) |
BR (1) | BR112022008861A2 (pt) |
CA (1) | CA3156277A1 (pt) |
IL (1) | IL292605A (pt) |
MX (1) | MX2022005572A (pt) |
WO (1) | WO2021092513A1 (pt) |
Families Citing this family (3)
Publication number | Priority date | Publication date | Assignee | Title |
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CN113025618B (zh) * | 2019-12-24 | 2024-02-06 | 朗信启昇(苏州)生物制药有限公司 | 一种x连锁遗传性视网膜劈裂症的基因治疗方案与应用 |
KR20220139352A (ko) * | 2020-02-07 | 2022-10-14 | 인텔리아 테라퓨틱스, 인크. | 칼리크레인(klkb1) 유전자 편집을 위한 조성물 및 방법 |
CN116790559B (zh) * | 2023-07-07 | 2023-11-28 | 山东舜丰生物科技有限公司 | 一种融合HNH结构域的V型Cas酶及其应用 |
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CA3067872A1 (en) | 2017-07-31 | 2019-02-07 | Regeneron Pharmaceuticals, Inc. | Cas-transgenic mouse embryonic stem cells and mice and uses thereof |
EP3687581A1 (en) | 2017-09-29 | 2020-08-05 | Intellia Therapeutics, Inc. | Polynucleotides, compositions, and methods for genome editing |
-
2020
- 2020-11-07 WO PCT/US2020/059568 patent/WO2021092513A1/en active Application Filing
- 2020-11-07 KR KR1020227016494A patent/KR20220097414A/ko unknown
- 2020-11-07 MX MX2022005572A patent/MX2022005572A/es unknown
- 2020-11-07 CA CA3156277A patent/CA3156277A1/en active Pending
- 2020-11-07 CN CN202080077587.XA patent/CN114746125A/zh active Pending
- 2020-11-07 US US17/774,284 patent/US20230001019A1/en active Pending
- 2020-11-07 BR BR112022008861A patent/BR112022008861A2/pt not_active Application Discontinuation
- 2020-11-07 EP EP20817149.6A patent/EP4054651A1/en active Pending
- 2020-11-07 AU AU2020379046A patent/AU2020379046A1/en active Pending
- 2020-11-07 IL IL292605A patent/IL292605A/en unknown
- 2020-11-07 JP JP2022524995A patent/JP2022553573A/ja active Pending
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CN114746125A (zh) | 2022-07-12 |
US20230001019A1 (en) | 2023-01-05 |
IL292605A (en) | 2022-07-01 |
MX2022005572A (es) | 2022-06-09 |
WO2021092513A1 (en) | 2021-05-14 |
JP2022553573A (ja) | 2022-12-23 |
CA3156277A1 (en) | 2021-05-14 |
KR20220097414A (ko) | 2022-07-07 |
AU2020379046A1 (en) | 2022-04-21 |
EP4054651A1 (en) | 2022-09-14 |
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