CA3076163A1 - Functional nucleic acid molecule and use thereof - Google Patents

Functional nucleic acid molecule and use thereof Download PDF

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Publication number
CA3076163A1
CA3076163A1 CA3076163A CA3076163A CA3076163A1 CA 3076163 A1 CA3076163 A1 CA 3076163A1 CA 3076163 A CA3076163 A CA 3076163A CA 3076163 A CA3076163 A CA 3076163A CA 3076163 A1 CA3076163 A1 CA 3076163A1
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CA
Canada
Prior art keywords
ires
sequence
nucleic acid
functional nucleic
acid molecule
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CA3076163A
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English (en)
French (fr)
Inventor
Stefano GUSTINCICH
Silvia ZUCCHELLI
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Scuola Internazionale Superiore di Studi Avanzati SISSA
Fondazione Istituto Italiano di Tecnologia
Original Assignee
Scuola Internazionale Superiore di Studi Avanzati SISSA
Fondazione Istituto Italiano di Tecnologia
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Application filed by Scuola Internazionale Superiore di Studi Avanzati SISSA, Fondazione Istituto Italiano di Tecnologia filed Critical Scuola Internazionale Superiore di Studi Avanzati SISSA
Publication of CA3076163A1 publication Critical patent/CA3076163A1/en
Pending legal-status Critical Current

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    • 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)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (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)
CA3076163A 2017-09-20 2018-09-20 Functional nucleic acid molecule and use thereof Pending CA3076163A1 (en)

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 (1)

Publication Number Publication Date
CA3076163A1 true CA3076163A1 (en) 2019-03-28

Family

ID=61024873

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3076163A Pending CA3076163A1 (en) 2017-09-20 2018-09-20 Functional nucleic acid molecule and use thereof

Country Status (8)

Country Link
US (1) US11649456B2 (cg-RX-API-DMAC7.html)
EP (1) EP3684928A1 (cg-RX-API-DMAC7.html)
JP (1) JP7258031B2 (cg-RX-API-DMAC7.html)
KR (1) KR102677300B1 (cg-RX-API-DMAC7.html)
CN (1) CN111386344B (cg-RX-API-DMAC7.html)
CA (1) CA3076163A1 (cg-RX-API-DMAC7.html)
IT (1) IT201700105372A1 (cg-RX-API-DMAC7.html)
WO (1) WO2019058304A1 (cg-RX-API-DMAC7.html)

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
US20250270561A1 (en) * 2020-10-08 2025-08-28 Transine Therapeutics Limited Functional nucleic acid molecules
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
WO2024177429A1 (ko) * 2023-02-24 2024-08-29 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

Family Cites Families (12)

* Cited by examiner, † Cited by third party
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
JP4654033B2 (ja) 2002-10-23 2011-03-16 バークシス コーポレーション 効率的なプレ−トランス−スプライシング分子の同定のためのスクリーニング方法
US7696406B2 (en) 2003-07-03 2010-04-13 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
ES2855577T3 (es) 2011-03-30 2021-09-23 Transine Therapeutics Ltd Molécula de ácido nucleico funcional y uso de la misma
WO2013176772A1 (en) * 2012-05-25 2013-11-28 The Regents Of The University Of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
EP2922554B1 (en) 2012-11-26 2022-02-23 ModernaTX, Inc. Terminally modified rna
ES2786193T3 (es) * 2012-12-12 2020-10-09 Broad Inst Inc Modificación por tecnología genética y optimización de sistemas, métodos y composiciones enzimáticas mejorados para la manipulación de secuencias
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

Also Published As

Publication number Publication date
KR102677300B1 (ko) 2024-06-24
WO2019058304A1 (en) 2019-03-28
CN111386344B (zh) 2024-02-02
IT201700105372A1 (it) 2019-03-20
JP7258031B2 (ja) 2023-04-14
CN111386344A (zh) 2020-07-07
JP2020535845A (ja) 2020-12-10
US20200224197A1 (en) 2020-07-16
KR20200117975A (ko) 2020-10-14
EP3684928A1 (en) 2020-07-29
US11649456B2 (en) 2023-05-16

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