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
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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    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • 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
    • 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)
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 (https=)
EP (1) EP3684928A1 (https=)
JP (1) JP7258031B2 (https=)
KR (1) KR102677300B1 (https=)
CN (1) CN111386344B (https=)
CA (1) CA3076163A1 (https=)
IT (1) IT201700105372A1 (https=)
WO (1) WO2019058304A1 (https=)

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
CN116322791A (zh) 2020-09-24 2023-06-23 意大利技术基金会 经修饰的功能性核酸分子
CA3196633A1 (en) * 2020-10-08 2022-04-14 Scuola Internazionale Superiore Di Studi Avanzati 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
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

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
EP1579004B1 (en) 2002-10-23 2010-06-16 VIRxSYS Corporation Screening methods for identification of efficient pre-trans-splicing molecules
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
EP3792359A1 (en) 2011-03-30 2021-03-17 Transine Therapeutics Limited Functional nucleic acid molecule and use thereof
EP3241902B1 (en) * 2012-05-25 2018-02-28 The Regents of The University of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
WO2014081507A1 (en) 2012-11-26 2014-05-30 Moderna Therapeutics, Inc. Terminally modified rna
CA2894684A1 (en) * 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of improved crispr-cas systems, methods and enzyme compositions for sequence manipulation in eukaryotes
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
DK3350327T3 (en) * 2015-10-23 2019-01-21 Caribou Biosciences Inc CONSTRUCTED CRISPR CLASS-2-NUCLEIC ACID TARGETING-NUCLEIC ACID

Also Published As

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

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