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BR112018015815A2 - regulação da expressão gênica através do controle mediado por aplicação de ribozimas auto-cleavantes - Google Patents

regulação da expressão gênica através do controle mediado por aplicação de ribozimas auto-cleavantes

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Publication number
BR112018015815A2
BR112018015815A2 BR112018015815A BR112018015815A BR112018015815A2 BR 112018015815 A2 BR112018015815 A2 BR 112018015815A2 BR 112018015815 A BR112018015815 A BR 112018015815A BR 112018015815 A BR112018015815 A BR 112018015815A BR 112018015815 A2 BR112018015815 A2 BR 112018015815A2
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BR
Brazil
Prior art keywords
gene expression
self
regulation
response
cleaving ribozyme
Prior art date
Application number
BR112018015815A
Other languages
English (en)
Inventor
K Boyne Alex
J Volles Michael
F Danos Olivier
Zennou Veronique
Guo Xuecui
Original Assignee
Meiragtx Uk Ii Ltd
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 Meiragtx Uk Ii Ltd filed Critical Meiragtx Uk Ii Ltd
Publication of BR112018015815A2 publication Critical patent/BR112018015815A2/pt

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    • 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
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
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    • 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
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    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/115Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
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    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N15/86Viral vectors
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/12Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/12Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/121Hammerhead
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3519Fusion with another nucleic acid
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/50Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/52Vector systems having a special element relevant for transcription encoding ribozyme for self-inactivation

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  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
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  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

a invenção proporciona construções polinucleotídicas para a regulação da expressão gênica por modulação baseada em aptâmeros de ribozimas e métodos de uso das construções para regular a expressão de genes em resposta à presença ou ausência de um ligante que liga-se o aptâmero de auto-clivagem. a invenção proporciona ainda métodos para a produção e uso de riboswitches que diminuem a expressão do gene alvo em resposta a um ligante de aptâmero bem como riboswitches que aumentam a expressão de gene alvo em resposta a um ligante de aptâmero.
BR112018015815A 2016-02-02 2017-02-02 regulação da expressão gênica através do controle mediado por aplicação de ribozimas auto-cleavantes BR112018015815A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662290187P 2016-02-02 2016-02-02
US201662290209P 2016-02-02 2016-02-02
PCT/US2017/016303 WO2017136608A2 (en) 2016-02-02 2017-02-02 Regulation of gene expression via aptamer-mediated control of self-cleaving ribozymes

Publications (1)

Publication Number Publication Date
BR112018015815A2 true BR112018015815A2 (pt) 2019-01-02

Family

ID=59500184

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112018015815A BR112018015815A2 (pt) 2016-02-02 2017-02-02 regulação da expressão gênica através do controle mediado por aplicação de ribozimas auto-cleavantes

Country Status (24)

Country Link
US (2) US11512310B2 (pt)
EP (1) EP3411506B1 (pt)
JP (1) JP7222715B2 (pt)
KR (1) KR20180118650A (pt)
CN (1) CN109476697B (pt)
AU (1) AU2017213845B2 (pt)
BR (1) BR112018015815A2 (pt)
CA (1) CA3048625A1 (pt)
DK (1) DK3411506T3 (pt)
ES (1) ES2954929T3 (pt)
FI (1) FI3411506T3 (pt)
HR (1) HRP20231119T1 (pt)
HU (1) HUE063465T2 (pt)
IL (1) IL260842B2 (pt)
LT (1) LT3411506T (pt)
MX (1) MX2018009369A (pt)
MY (1) MY198399A (pt)
PH (1) PH12018501651A1 (pt)
PL (1) PL3411506T3 (pt)
PT (1) PT3411506T (pt)
RS (1) RS64585B1 (pt)
SG (1) SG11201806589UA (pt)
SI (1) SI3411506T1 (pt)
WO (1) WO2017136608A2 (pt)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2018227057B2 (en) * 2017-03-02 2022-10-20 MEIRAGTX, UK II Limited Regulation of gene expression by aptamer-modulated RNase P cleavage
US20230265441A1 (en) 2020-03-24 2023-08-24 Meiragtx Uk Ii Limited Aptamers That Bind Thiamine Analogs and Derivatives
CA3204046A1 (en) * 2021-01-05 2022-07-14 James M. Carothers Modular kinetically-controlled functional rna constructs and related compositions, systems, and methods
WO2022150773A2 (en) * 2021-01-11 2022-07-14 The Regents Of The University Of California Ribozyme-activated rna constructs and uses thereof
IL313463A (en) * 2021-12-15 2024-08-01 Meiragtx Uk Ii Ltd Small molecule aptamers and ligands
IL313462A (en) 2021-12-15 2024-08-01 Meiragtx Uk Ii Ltd Polycistronic expression of intestinal peptides
CN114908095A (zh) * 2022-06-09 2022-08-16 南开大学 一种基于核酶的靶向抑制靶标rna的方法和应用

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050136035A1 (en) 2003-06-03 2005-06-23 Derek Ko Cell specific replication-competent viral vectors comprising a self processing peptide cleavage site
CA2545697C (en) * 2003-11-14 2016-12-20 Richard Mulligan Self-cleaving ribozymes and uses thereof
ES2633565T3 (es) 2010-11-12 2017-09-22 The General Hospital Corporation ARN no codificantes asociados a polycomb
US9222093B2 (en) * 2011-06-30 2015-12-29 The University Of Hong Kong Two-way, portable riboswitch mediated gene expression control device

Also Published As

Publication number Publication date
PT3411506T (pt) 2023-09-12
JP2019503205A (ja) 2019-02-07
AU2017213845A1 (en) 2018-09-06
MX2018009369A (es) 2019-01-10
CN109476697A (zh) 2019-03-15
PL3411506T3 (pl) 2023-11-27
DK3411506T3 (da) 2023-09-25
EP3411506A2 (en) 2018-12-12
ES2954929T3 (es) 2023-11-27
MY198399A (en) 2023-08-28
IL260842A (pt) 2018-09-20
HRP20231119T1 (hr) 2023-12-22
WO2017136608A4 (en) 2017-10-19
WO2017136608A3 (en) 2017-08-24
KR20180118650A (ko) 2018-10-31
AU2017213845B2 (en) 2023-06-29
FI3411506T3 (fi) 2023-09-25
RS64585B1 (sr) 2023-10-31
WO2017136608A2 (en) 2017-08-10
IL260842B2 (en) 2023-07-01
HUE063465T2 (hu) 2024-01-28
LT3411506T (lt) 2023-09-11
EP3411506B1 (en) 2023-06-28
EP3411506A4 (en) 2019-10-09
US20230220382A1 (en) 2023-07-13
CA3048625A1 (en) 2017-08-10
US20200032253A1 (en) 2020-01-30
PH12018501651A1 (en) 2019-06-03
JP7222715B2 (ja) 2023-02-15
SG11201806589UA (en) 2018-09-27
US11512310B2 (en) 2022-11-29
IL260842B1 (en) 2023-03-01
CN109476697B (zh) 2022-08-23
SI3411506T1 (sl) 2023-11-30

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