ES3059720T3 - Regulation of gene expression through aptamer-modulated polyadenylation - Google Patents

Regulation of gene expression through aptamer-modulated polyadenylation

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Publication number
ES3059720T3
ES3059720T3 ES17748182T ES17748182T ES3059720T3 ES 3059720 T3 ES3059720 T3 ES 3059720T3 ES 17748182 T ES17748182 T ES 17748182T ES 17748182 T ES17748182 T ES 17748182T ES 3059720 T3 ES3059720 T3 ES 3059720T3
Authority
ES
Spain
Prior art keywords
aptamer
gene expression
polyadenylation
modulated
regulation
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.)
Active
Application number
ES17748182T
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English (en)
Inventor
Michael Volles
Olivier Danos
Xuecui Guo
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.)
Meiragtx Gene Regulation Ltd
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Meiragtx Gene Regulation 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.)
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Publication of ES3059720T3 publication Critical patent/ES3059720T3/es
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Classifications

    • 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
    • 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
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered 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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/115—Aptamers, 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
    • 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
    • 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
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • 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
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/16—Aptamers
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Developing Agents For Electrophotography (AREA)

Abstract

La invención proporciona construcciones de polinucleótidos para la regulación de la expresión génica mediante la modulación, basada en aptámeros, de la supresión de la poliadenilación mediada por la ribonucleoproteína nuclear pequeña U1 (snRNP), así como métodos para utilizar dichas construcciones con el fin de regular la expresión génica en respuesta a la presencia o ausencia de un ligando que se une al aptámero. La construcción de polinucleótido contiene un sitio de unión a U1 en el contexto de un riboswitch que comprende una región efectora y un aptámero, de manera que cuando el aptámero se une a un ligando, se produce la expresión del gen diana. (Traducción automática con Google Translate, sin valor legal)
ES17748182T 2016-02-02 2017-02-02 Regulation of gene expression through aptamer-modulated polyadenylation Active ES3059720T3 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662290200P 2016-02-02 2016-02-02
PCT/US2017/016279 WO2017136591A1 (en) 2016-02-02 2017-02-02 Regulation of gene expression through aptamer-modulated polyadenylation

Publications (1)

Publication Number Publication Date
ES3059720T3 true ES3059720T3 (en) 2026-03-23

Family

ID=59500159

Family Applications (1)

Application Number Title Priority Date Filing Date
ES17748182T Active ES3059720T3 (en) 2016-02-02 2017-02-02 Regulation of gene expression through aptamer-modulated polyadenylation

Country Status (14)

Country Link
US (2) US11697815B2 (es)
EP (1) EP3411081B1 (es)
JP (1) JP7184649B2 (es)
KR (1) KR102856245B1 (es)
CN (1) CN109511259B (es)
AU (1) AU2017213835C1 (es)
EA (1) EA201891721A1 (es)
ES (1) ES3059720T3 (es)
IL (1) IL260840B2 (es)
MX (1) MX2018009370A (es)
MY (1) MY197192A (es)
PH (1) PH12018501650B1 (es)
SG (1) SG11201806605RA (es)
WO (1) WO2017136591A1 (es)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2769991C2 (ru) * 2015-11-12 2022-04-12 Байлор Колледж Оф Медсин Экзогенный контроль экспрессии генов млекопитающих с помощью опосредованной аптамерами модуляции полиаденилирования
KR102879552B1 (ko) 2020-03-24 2025-11-10 메이라지티엑스 진 레귤레이션 리미티드 티아민 유사체 및 유도체를 결합하는 압타머
WO2022015513A2 (en) 2020-07-13 2022-01-20 The Board Of Trustees Of The Leland Stanford Junior University Systems and methods to assess rna stability
US11492611B2 (en) * 2020-08-31 2022-11-08 The Board Of Trustees Of The Leland Stanford Junior University Systems and methods for producing RNA constructs with increased translation and stability
CN118434758A (zh) 2021-12-15 2024-08-02 梅里特斯英国第二有限公司 肠肽的多顺反子表达
AU2024305450A1 (en) 2023-06-14 2026-01-22 MeiraGTx Gene Regulation Limited Small molecule ligands and aptamers

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6692910B2 (en) 1997-07-28 2004-02-17 The University Of Connecticut Inhibition of a target messenger RNA with a modified U1 small nuclear RNA
US7563601B1 (en) * 2005-06-01 2009-07-21 City Of Hope Artificial riboswitch for controlling pre-mRNA splicing
GB0701722D0 (en) 2007-01-30 2007-03-07 Scottish Crop Res Inst Inhibition of gene expression
US9441221B2 (en) 2007-03-30 2016-09-13 Rutgers, The State University Of New Jersey Compositions and methods for gene silencing
EP2142672B1 (en) * 2007-03-30 2012-09-05 Rutgers, The State University of New Jersey Compositions and methods for gene silencing
US20120107331A1 (en) 2009-05-15 2012-05-03 Yale University Gemm riboswitches, structure-based compound design with gemm riboswitches, and methods and compositions for use of and with gemm riboswitches
US9125931B2 (en) 2010-04-06 2015-09-08 Massachusetts Institute Of Technology Post-transcriptional regulation of RNA-related processes using encoded protein-binding RNA aptamers
US11142762B2 (en) * 2012-07-05 2021-10-12 The Trustees Of The University Of Pennsylvania U1 snRNP regulates gene expression and modulates oncogenicity
PL3265563T3 (pl) 2015-02-02 2021-09-13 Meiragtx Uk Ii Limited Regulacja ekspresji genów poprzez modulację alternatywnego splicingu za pośrednictwem aptamerów
SMT202400091T1 (it) 2016-08-03 2024-05-14 Meiragtx Uk Ii Ltd Screening ad alta produzione basato su cellule per 5 aptameri

Also Published As

Publication number Publication date
JP7184649B2 (ja) 2022-12-06
US12559757B2 (en) 2026-02-24
AU2017213835B2 (en) 2024-02-01
KR20180117630A (ko) 2018-10-29
MY197192A (en) 2023-05-31
EP3411081C0 (en) 2026-01-07
PH12018501650B1 (en) 2024-07-03
CN109511259A (zh) 2019-03-22
SG11201806605RA (en) 2018-09-27
JP2019503204A (ja) 2019-02-07
EP3411081A1 (en) 2018-12-12
IL260840A (es) 2018-09-20
NZ745492A (en) 2025-03-28
EP3411081B1 (en) 2026-01-07
US20200017858A1 (en) 2020-01-16
KR102856245B1 (ko) 2025-09-05
PH12018501650A1 (en) 2019-06-03
IL260840B1 (en) 2025-08-01
CA3048620A1 (en) 2017-08-10
WO2017136591A1 (en) 2017-08-10
EA201891721A1 (ru) 2019-02-28
IL260840B2 (en) 2025-12-01
US20240191239A1 (en) 2024-06-13
BR112018015819A2 (pt) 2019-01-02
CN109511259B (zh) 2023-10-20
US11697815B2 (en) 2023-07-11
EP3411081A4 (en) 2019-12-11
AU2017213835A1 (en) 2018-09-06
MX2018009370A (es) 2018-11-12
AU2017213835C1 (en) 2024-04-18

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