IT201700105372A1 - Molecola di acido nucleico funzionale e relativo uso - Google Patents

Molecola di acido nucleico funzionale e relativo uso

Info

Publication number
IT201700105372A1
IT201700105372A1 IT102017000105372A IT201700105372A IT201700105372A1 IT 201700105372 A1 IT201700105372 A1 IT 201700105372A1 IT 102017000105372 A IT102017000105372 A IT 102017000105372A IT 201700105372 A IT201700105372 A IT 201700105372A IT 201700105372 A1 IT201700105372 A1 IT 201700105372A1
Authority
IT
Italy
Prior art keywords
nucleic acid
acid molecule
functional nucleic
functional
molecule
Prior art date
Application number
IT102017000105372A
Other languages
English (en)
Italian (it)
Inventor
Stefano Gustincich
Silvia Zucchelli
Original Assignee
Fondazione St Italiano Tecnologia
Scuola Int Superiore Di Studi Avanzati Sissa
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Fondazione St Italiano Tecnologia, Scuola Int Superiore Di Studi Avanzati Sissa filed Critical Fondazione St Italiano Tecnologia
Priority to IT102017000105372A priority Critical patent/IT201700105372A1/it
Priority to US16/647,721 priority patent/US11649456B2/en
Priority to JP2020537887A priority patent/JP7258031B2/ja
Priority to CA3076163A priority patent/CA3076163A1/en
Priority to KR1020207011322A priority patent/KR102677300B1/ko
Priority to PCT/IB2018/057262 priority patent/WO2019058304A1/en
Priority to EP18782521.1A priority patent/EP3684928A1/en
Priority to CN201880074261.4A priority patent/CN111386344B/zh
Publication of IT201700105372A1 publication Critical patent/IT201700105372A1/it

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67General methods for enhancing the expression
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3519Fusion with another nucleic acid
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2330/00Production
    • C12N2330/50Biochemical production, i.e. in a transformed host cell
    • C12N2330/51Specially adapted vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00Vectors comprising a special translation-regulating system
    • C12N2840/10Vectors comprising a special translation-regulating system regulates levels of translation
    • C12N2840/105Vectors comprising a special translation-regulating system regulates levels of translation enhancing translation
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00Vectors comprising a special translation-regulating system
    • C12N2840/20Vectors comprising a special translation-regulating system translation of more than one cistron
    • C12N2840/203Vectors 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)
IT102017000105372A 2017-09-20 2017-09-20 Molecola di acido nucleico funzionale e relativo uso IT201700105372A1 (it)

Priority Applications (8)

Application Number Priority Date Filing Date Title
IT102017000105372A IT201700105372A1 (it) 2017-09-20 2017-09-20 Molecola di acido nucleico funzionale e relativo uso
US16/647,721 US11649456B2 (en) 2017-09-20 2018-09-20 Functional nucleic acid molecule and use thereof
JP2020537887A JP7258031B2 (ja) 2017-09-20 2018-09-20 機能性核酸分子及びその使用
CA3076163A CA3076163A1 (en) 2017-09-20 2018-09-20 Functional nucleic acid molecule and use thereof
KR1020207011322A KR102677300B1 (ko) 2017-09-20 2018-09-20 기능성 핵산 분자 및 그의 용도
PCT/IB2018/057262 WO2019058304A1 (en) 2017-09-20 2018-09-20 FUNCTIONAL NUCLEIC ACID MOLECULE AND ASSOCIATED APPLICATIONS
EP18782521.1A EP3684928A1 (en) 2017-09-20 2018-09-20 Functional nucleic acid molecule and use thereof
CN201880074261.4A CN111386344B (zh) 2017-09-20 2018-09-20 功能性核酸分子及其用途

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
IT102017000105372A IT201700105372A1 (it) 2017-09-20 2017-09-20 Molecola di acido nucleico funzionale e relativo uso

Publications (1)

Publication Number Publication Date
IT201700105372A1 true IT201700105372A1 (it) 2019-03-20

Family

ID=61024873

Family Applications (1)

Application Number Title Priority Date Filing Date
IT102017000105372A IT201700105372A1 (it) 2017-09-20 2017-09-20 Molecola di acido nucleico funzionale e relativo uso

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)

* Cited by examiner, † Cited by third party
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 (1)

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WO2012133947A1 (en) * 2011-03-30 2012-10-04 Riken Functional nucleic acid molecule and use thereof

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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
HRP20220607T1 (hr) 2012-11-26 2022-06-24 Modernatx, Inc. Terminalno modificirana rna
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
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PATRUCCO LAURA ET AL: "Engineering mammalian cell factories with SINEUP noncoding RNAs to improve translation of secreted proteins", 2 June 2015, GENE, ELSEVIER, AMSTERDAM, NL, PAGE(S) 287 - 293, ISSN: 0378-1119, XP029247971 *

Also Published As

Publication number Publication date
CN111386344B (zh) 2024-02-02
JP2020535845A (ja) 2020-12-10
WO2019058304A1 (en) 2019-03-28
CA3076163A1 (en) 2019-03-28
CN111386344A (zh) 2020-07-07
US20200224197A1 (en) 2020-07-16
KR102677300B1 (ko) 2024-06-24
EP3684928A1 (en) 2020-07-29
US11649456B2 (en) 2023-05-16
KR20200117975A (ko) 2020-10-14
JP7258031B2 (ja) 2023-04-14

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