SG11201907700XA - Regulation of gene expression by aptamer-mediated accessibility of polyadenylation signals - Google Patents

Regulation of gene expression by aptamer-mediated accessibility of polyadenylation signals

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Publication number
SG11201907700XA
SG11201907700XA SG11201907700XA SG11201907700XA SG11201907700XA SG 11201907700X A SG11201907700X A SG 11201907700XA SG 11201907700X A SG11201907700X A SG 11201907700XA SG 11201907700X A SG11201907700X A SG 11201907700XA SG 11201907700X A SG11201907700X A SG 11201907700XA
Authority
SG
Singapore
Prior art keywords
aptamer
international
gene expression
rule
ligand
Prior art date
Application number
SG11201907700XA
Inventor
Xuecui Guo
Joonhee Han
Zhaojing Zhong
Original Assignee
Meiragtx Uk Ii Limited
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 date
Application filed by Meiragtx Uk Ii Limited filed Critical Meiragtx Uk Ii Limited
Publication of SG11201907700XA publication Critical patent/SG11201907700XA/en

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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
    • 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
    • C12N15/115Aptamers, 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
    • 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
    • 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
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • 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
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/16Aptamers
    • 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
    • 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

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  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Management, Administration, Business Operations System, And Electronic Commerce (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 30 August 2018 (30.08.2018) W I PO I P CT 0111111010 0111 °nolo OH lull um° imio oimIE (10) International Publication Number WO 2018/156658 Al (51) International Patent Classification: A61K 48/00 (2006.01) C12N 15/115 (2010.01) C12N 15/113 (2010.01) (21) International Application Number: PCT/US2018/019056 (22) International Filing Date: 21 February 2018 (21.02.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/461,689 21 February 2017 (21.02.2017) US (71) Applicant: MEIRAGTX, UK II Limited [GB/GB]; 92 Britannia Walk, London, N17NQ, United Kingdom (GB). (72) Inventors: GUO, Xuecui; 1 Bungtown Road, Cold Spring Harbor, NY 11724 (US). HAN, Joonhee; 70 Christopher Columbus Dr., Apt. 704, Jersey City, NJ 07302 (US). ZHONG, Zhaojing; 1516 Seminole Street, Bronx, NY 10461 (US). (74) Agent: SOMERVILLE, Deborah, A. et al.; Fox Roth- schild LLP, Princeton Pike Corporate Center, 997 Lenox Drive, Bldg. 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, JO, 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, (54) Title: REGULATION OF GENE EXPRESSION BY APTAMER-MEDIATED ACCESSIBILITY OF POLYADENYLATION SIGNALS Stem-loop Structure Ligand-free DSE 3' UTR with PolyA Sequence nn Gene LI Expression —I CDS Ligand-bound Aptamer Ligand aaja0 A = AATAAA DS E Gene Expression CDS 3' UTR with PolyA Sequence FIG. 1 b (57) : The invention provides polynucleotide constructs for the regulation of gene expression by aptamer-based modulation of the accessibility of one or more polyadenylation signals 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 contains a riboswitch comprising an aptamer and an effector stem loop, wherein the effector stem loop comprises a polyadenylation signal sequence. Aptanner [Continued on next page] WO 2018/156658 Al O II TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). Declarations under Rule 4.17: as to applicant's entitlement to apply for and be granted a patent (Rule 4.17(11)) as to the applicant's entitlement to claim the priority of the earlier application (Rule 4.17(iii)) Published: with international search report (Art. 21(3)) with sequence listing part of description (Rule 5.2(a))
SG11201907700XA 2017-02-21 2018-02-21 Regulation of gene expression by aptamer-mediated accessibility of polyadenylation signals SG11201907700XA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762461689P 2017-02-21 2017-02-21
PCT/US2018/019056 WO2018156658A1 (en) 2017-02-21 2018-02-21 Regulation of gene expression by aptamer-mediated accessibility of polyadenylation signals

Publications (1)

Publication Number Publication Date
SG11201907700XA true SG11201907700XA (en) 2019-09-27

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Family Applications (1)

Application Number Title Priority Date Filing Date
SG11201907700XA SG11201907700XA (en) 2017-02-21 2018-02-21 Regulation of gene expression by aptamer-mediated accessibility of polyadenylation signals

Country Status (14)

Country Link
US (2) US20200010836A1 (en)
EP (1) EP3585443A4 (en)
JP (1) JP7139345B2 (en)
KR (1) KR102645079B1 (en)
CN (1) CN110636868B (en)
AU (1) AU2018224048A1 (en)
BR (1) BR112019017444A2 (en)
CA (1) CA3054294A1 (en)
EA (1) EA201991928A1 (en)
IL (1) IL268819B1 (en)
MX (1) MX2019009972A (en)
NZ (1) NZ756568A (en)
SG (1) SG11201907700XA (en)
WO (1) WO2018156658A1 (en)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20230265441A1 (en) 2020-03-24 2023-08-24 Meiragtx Uk Ii Limited Aptamers That Bind Thiamine Analogs and Derivatives
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
US5567588A (en) 1990-06-11 1996-10-22 University Research Corporation Systematic evolution of ligands by exponential enrichment: Solution SELEX
US5580737A (en) 1990-06-11 1996-12-03 Nexstar Pharmaceuticals, Inc. High-affinity nucleic acid ligands that discriminate between theophylline and caffeine
JP2763958B2 (en) 1990-06-11 1998-06-11 ネクスター ファーマスーティカルズ,インコーポレイテッド Nucleic acid ligand
US5270163A (en) 1990-06-11 1993-12-14 University Research Corporation Methods for identifying nucleic acid ligands
US20090117545A1 (en) * 2004-10-07 2009-05-07 Breaker Ronald R Glycine riboswitches, methods for their use, and compositions for use with glycine riboswitches Cross-Reference to Related Applications
KR20100017893A (en) * 2007-05-29 2010-02-16 예일 유니버시티 Methods and compositions related to riboswitches that control alternative splicing and rna processing
CA2764313A1 (en) * 2009-06-02 2010-12-09 Biorelix, Inc. Methods for screening and identifying compounds
PT3265563T (en) * 2015-02-02 2021-06-03 Meiragtx Uk Ii Ltd Regulation of gene expression by aptamer-mediated modulation of alternative splicing
FI3430134T3 (en) 2015-06-18 2023-01-13 Novel crispr enzymes and systems
WO2018025085A2 (en) 2016-08-03 2018-02-08 Meiragtx Uk Ii Limited High throughput cell-based screening for aptamers

Also Published As

Publication number Publication date
US20200010836A1 (en) 2020-01-09
CA3054294A1 (en) 2018-08-30
NZ756568A (en) 2024-03-22
MX2019009972A (en) 2020-01-15
US20240093209A1 (en) 2024-03-21
BR112019017444A2 (en) 2020-04-14
EP3585443A1 (en) 2020-01-01
EP3585443A4 (en) 2020-12-16
JP7139345B2 (en) 2022-09-20
KR20190126334A (en) 2019-11-11
IL268819A (en) 2019-10-31
CN110636868A (en) 2019-12-31
EA201991928A1 (en) 2020-01-09
IL268819B1 (en) 2024-03-01
AU2018224048A1 (en) 2019-09-12
WO2018156658A1 (en) 2018-08-30
KR102645079B1 (en) 2024-03-08
JP2020508057A (en) 2020-03-19
CN110636868B (en) 2024-03-15

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