Patent SG-11201907700X-A (SG)
Title: Regulation of gene expression by aptamer-mediated accessibility of polyadenylation signals
Inventor: GUO XUECUI
IPC: C12N15/113
Filed: 2018-02-21
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))