SG11201806605RA - Regulation of gene expression through aptamer-modulated polyadenylation - Google Patents

Regulation of gene expression through aptamer-modulated polyadenylation

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Publication number
SG11201806605RA
SG11201806605RA SG11201806605RA SG11201806605RA SG11201806605RA SG 11201806605R A SG11201806605R A SG 11201806605RA SG 11201806605R A SG11201806605R A SG 11201806605RA SG 11201806605R A SG11201806605R A SG 11201806605RA SG 11201806605R A SG11201806605R A SG 11201806605RA
Authority
SG
Singapore
Prior art keywords
aptamer
international
gene expression
rule
polyadenylation
Prior art date
Application number
SG11201806605RA
Inventor
Michael Volles
Olivier Danos
Xuecui Guo
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 SG11201806605RA publication Critical patent/SG11201806605RA/en

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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
    • C12N15/79—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
    • C12N15/11—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
    • 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
    • C12N15/63—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
    • 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

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  • 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)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (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

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (10) International Publication Number (43) International Publication Date WO 2017/136591 Al 10 August 2017 (10.08.2017) WIPO I PCT 111111 11111111 0 111011111111111 010 11111 01110111 0111111111111111101111111111111 0111111 (51) International Patent Classification: A61K 48/00 (2006.01) CO7H 21/04 (2006.01) C12N 15/86 (2006.01) (21) International Application Number: PCT/US2017/016279 (22) International Filing Date: 2 February 2017 (02.02.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/290,200 2 February 2016 (02.02.2016) US (71) Applicants: MEIRAGTX UK II LIMITED [GB/GB]; 92 Britannia Walk, London, N1 7NQ (GB). MEIRAGTX, LLC [US/US]; 450 East 29th Street 15th Floor, New York, NY 10016 (US). (72) Inventors: VOLLES, Michael, J.; 69 Bolton Street Apt. 102, Cambridge, MA 02140 (US). DANOS, Olivier, F.; 49 Hancock Street, Boston, MA 02114 (US). GUO, Xuecui; 1 Bungtown Road, Cold Spring Harbor, NY 11724 (US). (74) Agents: SOMERVILLE, Deborah, A. et al.; Fox Roth- schild, LLP, Princeton Pike Corporate Center, 997 Lenox Drive, Building 3, Lawrenceville, NJ 08648-2311 (US). (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. (84) Designated States (unless otherwise indicated, for every kind of regional protection available): ARIPO (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). arations under Rule 4.17: as to applicant's entitlement to apply for and be granted a patent (Rule 4.1700) as to the applicant's entitlement to claim the priority of the earlier application (Rule 4.17(iii)) ished: with international search report (Art. 21(3)) before the expiration of the time limit for amending the claims and to be republished in the event of receipt of amendments (Rule 48.2(h)) Decl Publ W O 20 17 / 1365 9 1 Al (54) Title: REGULATION OF GENE EXPRESSION THROUGH APTAMER-MODULATED POLYADENYLATION (57) : The invention provides polynucleotide constructs for the regulation of gene expression by aptamer-based modulation of Ul small nuclear ribonucleoprotein (snRNP)-mediated suppression of polyadenylation and methods of using the constructs to regulate gene expression in response to the presence or absence of a ligand that binds the aptamer. The polynucleotide construct con - tains a Ul binding site in the context of a riboswitch comprising an effector region and an aptamer such that when the aptamer binds a ligand, target gene expression occurs.
SG11201806605RA 2016-02-02 2017-02-02 Regulation of gene expression through aptamer-modulated polyadenylation SG11201806605RA (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
SG11201806605RA true SG11201806605RA (en) 2018-09-27

Family

ID=59500159

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11201806605RA SG11201806605RA (en) 2016-02-02 2017-02-02 Regulation of gene expression through aptamer-modulated polyadenylation

Country Status (14)

Country Link
US (1) US11697815B2 (en)
EP (1) EP3411081A4 (en)
JP (1) JP7184649B2 (en)
KR (1) KR20180117630A (en)
CN (1) CN109511259B (en)
AU (1) AU2017213835C1 (en)
BR (1) BR112018015819A2 (en)
CA (1) CA3048620A1 (en)
EA (1) EA201891721A1 (en)
MX (1) MX2018009370A (en)
MY (1) MY197192A (en)
PH (1) PH12018501650A1 (en)
SG (1) SG11201806605RA (en)
WO (1) WO2017136591A1 (en)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2969015T3 (en) * 2015-11-12 2024-05-16 Baylor College Medicine Exogenous control of mammalian gene expression by modulating aptamer-mediated polyadenylation
MX2022011940A (en) 2020-03-24 2023-01-05 Meiragtx Uk Ii Ltd Aptamers that bind thiamine analogs and derivatives.
EP4179112A2 (en) 2020-07-13 2023-05-17 The Board of Trustees of the Leland Stanford Junior University Systems and methods to assess rna stability
WO2022047427A2 (en) * 2020-08-31 2022-03-03 The Board Of Trustees Of The Leland Stanford Junior University Systems and methods for producing rna constructs with increased translation and stability
WO2023131811A2 (en) 2021-12-15 2023-07-13 Meiragtx Uk Ii Limited Polycistronic expression of gut peptides

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
WO2010132665A1 (en) * 2009-05-15 2010-11-18 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
EP3699284A1 (en) * 2012-07-05 2020-08-26 The Trustees of the University of Pennsylvania U1 snrnp regulates gene expression and modulates oncogenicity
EA037120B1 (en) * 2015-02-02 2021-02-09 МЕИРЭДжТиЭкс ЮКей II ЛИМИТЕД Regulation of gene expression by aptamer-mediated modulation of alternative splicing
RS65228B1 (en) * 2016-08-03 2024-03-29 Meiragtx Uk Ii Ltd High throughput cell-based screening for aptamers

Also Published As

Publication number Publication date
AU2017213835A1 (en) 2018-09-06
PH12018501650A1 (en) 2019-06-03
CA3048620A1 (en) 2017-08-10
JP2019503204A (en) 2019-02-07
CN109511259A (en) 2019-03-22
MY197192A (en) 2023-05-31
AU2017213835C1 (en) 2024-04-18
AU2017213835B2 (en) 2024-02-01
EA201891721A1 (en) 2019-02-28
JP7184649B2 (en) 2022-12-06
EP3411081A1 (en) 2018-12-12
MX2018009370A (en) 2018-11-12
US20200017858A1 (en) 2020-01-16
EP3411081A4 (en) 2019-12-11
WO2017136591A1 (en) 2017-08-10
CN109511259B (en) 2023-10-20
KR20180117630A (en) 2018-10-29
US11697815B2 (en) 2023-07-11
BR112018015819A2 (en) 2019-01-02

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