KR102677300B1 - 기능성 핵산 분자 및 그의 용도 - Google Patents
기능성 핵산 분자 및 그의 용도 Download PDFInfo
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- KR102677300B1 KR102677300B1 KR1020207011322A KR20207011322A KR102677300B1 KR 102677300 B1 KR102677300 B1 KR 102677300B1 KR 1020207011322 A KR1020207011322 A KR 1020207011322A KR 20207011322 A KR20207011322 A KR 20207011322A KR 102677300 B1 KR102677300 B1 KR 102677300B1
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Classifications
-
- 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
-
- 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
- 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
- C12N15/67—General methods for enhancing the expression
-
- 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
-
- 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
- C12N2310/11—Antisense
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3519—Fusion with another nucleic acid
-
- 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
- C12N2330/00—Production
- C12N2330/50—Biochemical production, i.e. in a transformed host cell
- C12N2330/51—Specially adapted vectors
-
- 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
- C12N2840/00—Vectors comprising a special translation-regulating system
- C12N2840/10—Vectors comprising a special translation-regulating system regulates levels of translation
- C12N2840/105—Vectors comprising a special translation-regulating system regulates levels of translation enhancing translation
-
- 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
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| IT102017000105372A IT201700105372A1 (it) | 2017-09-20 | 2017-09-20 | Molecola di acido nucleico funzionale e relativo uso |
| IT102017000105372 | 2017-09-20 | ||
| PCT/IB2018/057262 WO2019058304A1 (en) | 2017-09-20 | 2018-09-20 | FUNCTIONAL NUCLEIC ACID MOLECULE AND ASSOCIATED APPLICATIONS |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| KR20200117975A KR20200117975A (ko) | 2020-10-14 |
| KR102677300B1 true KR102677300B1 (ko) | 2024-06-24 |
Family
ID=61024873
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020207011322A Active KR102677300B1 (ko) | 2017-09-20 | 2018-09-20 | 기능성 핵산 분자 및 그의 용도 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US11649456B2 (enExample) |
| EP (1) | EP3684928A1 (enExample) |
| JP (1) | JP7258031B2 (enExample) |
| KR (1) | KR102677300B1 (enExample) |
| CN (1) | CN111386344B (enExample) |
| CA (1) | CA3076163A1 (enExample) |
| IT (1) | IT201700105372A1 (enExample) |
| WO (1) | WO2019058304A1 (enExample) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IT201900011490A1 (it) | 2019-07-11 | 2021-01-11 | Scuola Int Superiore Di Studi Avanzati Sissa | MOLECOLE DI ACIDO NUCLEICO FUNZIONALI CHE AUMENTANO LA TRADUZIONE DI UN mRNA DELLA FRATASSINA |
| CN110484563B (zh) * | 2019-07-25 | 2023-04-07 | 新乡医学院 | 哺乳动物细胞组合表达载体、表达系统、制备方法和应用 |
| JP2023542529A (ja) * | 2020-09-24 | 2023-10-10 | フォンダジオネ イスティツト イタリアノ ディ テクノロジア | 修飾された機能性核酸分子 |
| JP2023545083A (ja) * | 2020-10-08 | 2023-10-26 | トランシネ セラピューティクス リミテッド | 機能性核酸分子 |
| EP3992289A1 (en) | 2020-10-30 | 2022-05-04 | Transine Therapeutics Limited | Functional nucleic acid molecules incorporating protein binding domain |
| CN112126644B (zh) * | 2020-11-25 | 2021-03-12 | 北京立康生命科技有限公司 | 一种非依赖帽结构下进行细胞外转录的mRNA、呈递细胞及应用 |
| EP4063505A1 (en) | 2021-03-22 | 2022-09-28 | Fondazione Istituto Italiano di Tecnologia | Functional nucleic acid molecules directed to targets for nervous system disorders |
| GB202205423D0 (en) | 2022-04-12 | 2022-05-25 | Transine Therapeutics Ltd | Functional nucleic acid molecule |
| GB202207796D0 (en) | 2022-05-26 | 2022-07-13 | Fondazione St Italiano Tecnologia | Functional nucleic acid molecule |
| GB202207795D0 (en) | 2022-05-26 | 2022-07-13 | Fondazione St Italiano Tecnologia | Functional nucleic acid molecule |
| CN116004696B (zh) * | 2023-02-01 | 2024-03-29 | 郑州贝贝生物科技有限公司 | 可与IRES组合的3ˊUTR加茎环结构基因及其应用、mRNA表达系统 |
| KR20240132212A (ko) * | 2023-02-24 | 2024-09-03 | 서울시립대학교 산학협력단 | DNA 템플릿 및 이를 이용하여 제조된 mRNA 백신 |
| CN117947028A (zh) * | 2023-12-29 | 2024-04-30 | 北京衡昱生物科技有限公司 | Ires-w3-31序列及其应用 |
Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2012133947A1 (en) | 2011-03-30 | 2012-10-04 | Riken | Functional nucleic acid molecule and use thereof |
| WO2014081507A1 (en) | 2012-11-26 | 2014-05-30 | Moderna Therapeutics, Inc. | Terminally modified rna |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6869779B1 (en) * | 1998-08-27 | 2005-03-22 | Fuso Pharmaceutical Industries, Ltd. | Nucleic acid sequence for potentiating the expression of useful gene and method therefor |
| US7468275B2 (en) * | 2000-01-28 | 2008-12-23 | The Scripps Research Institute | Synthetic internal ribosome entry sites and methods of identifying same |
| EP1579004B1 (en) * | 2002-10-23 | 2010-06-16 | VIRxSYS Corporation | Screening methods for identification of efficient pre-trans-splicing molecules |
| WO2005019449A2 (en) * | 2003-07-03 | 2005-03-03 | Board Of Trustees Operating Michigan State University | Expression of a recombinant transgene |
| TWI300441B (en) * | 2005-09-21 | 2008-09-01 | Univ Chung Yuan Christian | A polynucleotide with ires activity |
| KR101161622B1 (ko) * | 2009-08-31 | 2012-07-04 | 헬릭스 주식회사 | 번역 효율 증진용 ｄｎａ 단편 및 이를 포함하는 재조합 벡터 |
| RS59199B1 (sr) * | 2012-05-25 | 2019-10-31 | Univ California | Metode i jedinjenja za rnk-upravljanu ciljanu dnk modifikaciju i za rnk- upravljanu modulaciju transkripta |
| EP2898075B1 (en) * | 2012-12-12 | 2016-03-09 | The Broad Institute, Inc. | Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation |
| CN105624156B (zh) * | 2014-11-04 | 2021-07-16 | 清华大学 | 含有反向sineb2重复序列的人工非编码rna及其在增强靶蛋白翻译中的用途 |
| WO2017070598A1 (en) * | 2015-10-23 | 2017-04-27 | Caribou Biosciences, Inc. | Engineered crispr class 2 cross-type nucleic-acid targeting nucleic acids |
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Patent Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2012133947A1 (en) | 2011-03-30 | 2012-10-04 | Riken | Functional nucleic acid molecule and use thereof |
| WO2014081507A1 (en) | 2012-11-26 | 2014-05-30 | Moderna Therapeutics, Inc. | Terminally modified rna |
| JP2015535430A (ja) | 2012-11-26 | 2015-12-14 | モデルナ セラピューティクス インコーポレイテッドＭｏ | 末端修飾ｒｎａ |
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| JP2020535845A (ja) | 2020-12-10 |
| WO2019058304A1 (en) | 2019-03-28 |
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| CN111386344A (zh) | 2020-07-07 |
| IT201700105372A1 (it) | 2019-03-20 |
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| US11649456B2 (en) | 2023-05-16 |
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| JP7258031B2 (ja) | 2023-04-14 |
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