BR112023026961A2 - Composições e métodos para modular a expressão de genes - Google Patents
Composições e métodos para modular a expressão de genesInfo
- Publication number
- BR112023026961A2 BR112023026961A2 BR112023026961A BR112023026961A BR112023026961A2 BR 112023026961 A2 BR112023026961 A2 BR 112023026961A2 BR 112023026961 A BR112023026961 A BR 112023026961A BR 112023026961 A BR112023026961 A BR 112023026961A BR 112023026961 A2 BR112023026961 A2 BR 112023026961A2
- Authority
- BR
- Brazil
- Prior art keywords
- compositions
- rna
- methods
- gene expression
- expression
- Prior art date
Links
- 230000014509 gene expression Effects 0.000 title abstract 5
- 239000000203 mixture Substances 0.000 title abstract 4
- 108091032973 (ribonucleotides)n+m Proteins 0.000 abstract 5
- 108091028043 Nucleic acid sequence Proteins 0.000 abstract 3
- 108090000623 proteins and genes Proteins 0.000 abstract 3
- 230000002068 genetic effect Effects 0.000 abstract 2
- 230000028993 immune response Effects 0.000 abstract 2
- 102000040650 (ribonucleotides)n+m Human genes 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 210000005260 human cell Anatomy 0.000 abstract 1
- 108091033319 polynucleotide Proteins 0.000 abstract 1
- 102000040430 polynucleotide Human genes 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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- 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
- C12N15/1138—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 against receptors or cell surface proteins
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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
- C07K14/575—Hormones
- C07K14/65—Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
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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
- A61P17/00—Drugs for dermatological disorders
- A61P17/02—Drugs for dermatological disorders for treating wounds, ulcers, burns, scars, keloids, or the like
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P17/00—Drugs for dermatological disorders
- A61P17/06—Antipsoriatics
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
- A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
- A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
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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
- C07K14/52—Cytokines; Lymphokines; Interferons
- C07K14/54—Interleukins [IL]
- C07K14/5406—IL-4
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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
- C12N15/1136—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 against growth factors, growth regulators, cytokines, lymphokines or hormones
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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
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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)
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/335—Modified T or U
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2840/00—Vectors comprising a special translation-regulating system
- C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
- C12N2840/203—Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/03—Phosphoric monoester hydrolases (3.1.3)
- C12Y301/03001—Alkaline phosphatase (3.1.3.1)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Biophysics (AREA)
- General Engineering & Computer Science (AREA)
- Microbiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Endocrinology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Immunology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Diabetes (AREA)
- Dermatology (AREA)
- Epidemiology (AREA)
- Physical Education & Sports Medicine (AREA)
- Rheumatology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Transplantation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
composições e métodos para modular a expressão de genes. a presente invenção se refere a composições e métodos para modular a expressão de genes, compreendendo construtos de ácido polinucleico ou rna recombinante compreendendo uma primeira sequência de rna que codifica um gene de interesse, e uma segunda sequência de rna compreendendo pelo menos dois elementos genéticos que modulam a expressão de um ou mais rnas alvo. os construtos de rna recombinante descritos na presente invenção induzem uma resposta imune em uma célula humana que é mais baixa do que a resposta imune induzida por um construto de rna recombinante correspondente compreendendo a primeira sequência de rna que codifica um gene de interesse e uma segunda sequência de rna correspondente compreendendo no máximo um de pelo menos dois elementos genéticos. também é revelado na presente invenção o uso das composições no tratamento de doenças e na modulação de expressão de dois ou mais genes.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202163213829P | 2021-06-23 | 2021-06-23 | |
PCT/IB2022/000358 WO2022269356A2 (en) | 2021-06-23 | 2022-06-22 | Compositions and methods for modulating expression of genes |
Publications (1)
Publication Number | Publication Date |
---|---|
BR112023026961A2 true BR112023026961A2 (pt) | 2024-03-12 |
Family
ID=83546992
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112023026961A BR112023026961A2 (pt) | 2021-06-23 | 2022-06-22 | Composições e métodos para modular a expressão de genes |
Country Status (10)
Country | Link |
---|---|
US (1) | US20240117361A1 (pt) |
EP (1) | EP4359533A2 (pt) |
KR (1) | KR20240024172A (pt) |
CN (1) | CN117813380A (pt) |
AU (1) | AU2022299474A1 (pt) |
BR (1) | BR112023026961A2 (pt) |
CA (1) | CA3221277A1 (pt) |
IL (1) | IL308979A (pt) |
TW (1) | TW202309288A (pt) |
WO (1) | WO2022269356A2 (pt) |
Family Cites Families (7)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
JP2003529349A (ja) * | 2000-02-18 | 2003-10-07 | ロード アイランド ホスピタル, ア ライフスパン パートナー | 骨障害の治療 |
JP5078904B2 (ja) * | 2005-11-21 | 2012-11-21 | ジヨンソン・アンド・ジヨンソン・リサーチ・ピーテイワイ・リミテツド | 多標的干渉rnaならびにそれらの使用および設計方法 |
EP1938802A1 (en) * | 2006-12-22 | 2008-07-02 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Interfering RNAs targeting pro-inflammatory cytokines |
EP4006153A1 (en) * | 2018-01-12 | 2022-06-01 | Curocell Inc. | Enhanced immune cells using dual shrna and composition including the same |
EP3898982A2 (en) * | 2018-12-19 | 2021-10-27 | Versameb AG | Rna encoding a protein |
KR20220035326A (ko) * | 2019-05-02 | 2022-03-22 | 셀리아드 | 다중화된 억제성 rna를 갖는 세포 |
BR112022012324A2 (pt) * | 2019-12-23 | 2022-10-11 | Versameb Ag | Composições e métodos para modular simultaneamente expressão de genes |
-
2022
- 2022-06-22 WO PCT/IB2022/000358 patent/WO2022269356A2/en active Application Filing
- 2022-06-22 EP EP22778044.2A patent/EP4359533A2/en active Pending
- 2022-06-22 CA CA3221277A patent/CA3221277A1/en active Pending
- 2022-06-22 CN CN202280044943.7A patent/CN117813380A/zh active Pending
- 2022-06-22 KR KR1020247000488A patent/KR20240024172A/ko unknown
- 2022-06-22 IL IL308979A patent/IL308979A/en unknown
- 2022-06-22 BR BR112023026961A patent/BR112023026961A2/pt unknown
- 2022-06-22 AU AU2022299474A patent/AU2022299474A1/en active Pending
- 2022-06-23 TW TW111123540A patent/TW202309288A/zh unknown
-
2023
- 2023-12-18 US US18/542,975 patent/US20240117361A1/en active Pending
Also Published As
Publication number | Publication date |
---|---|
CA3221277A1 (en) | 2022-12-29 |
CN117813380A (zh) | 2024-04-02 |
AU2022299474A1 (en) | 2023-12-21 |
TW202309288A (zh) | 2023-03-01 |
US20240117361A1 (en) | 2024-04-11 |
WO2022269356A3 (en) | 2023-05-25 |
IL308979A (en) | 2024-01-01 |
WO2022269356A2 (en) | 2022-12-29 |
KR20240024172A (ko) | 2024-02-23 |
EP4359533A2 (en) | 2024-05-01 |
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