Patent SG-11201908043U-A (SG)
Title: Regulation of gene expression by aptamer-modulated rnase p cleavage
Inventor: GUO XUECUI
IPC: C12N15/86
Filed: 2018-03-02
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 07 September 2018 (07.09.2018) WIPO I PCT omit VIII °nolo mu olollm 1o11 ono oimIE (10) International Publication Number WO 2018/161053 Al (51) International Patent Classification: C12N 15/113 (2010.01) Cl 2N 15/86 (2006.01) C12N 15/11 (2006.01) Cl 2N 15/67(2006.01) C12N 15/115 (2010.01) C12N 15/861 (2006.01) C12N 15/63 (2006.01) Cl 2P 19/34 (2006.01) (21) International Application Number: PCT/US2018/020795 (22) International Filing Date: 02 March 2018 (02.03.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/466,138 02 March 2017 (02.03.2017) US (71) Applicant: MEIRAGTX, UK II LIMITED [GB/GB]; 92 Britannia Walk, London, N1 7NQ (GB). (72) Inventor: GUO, Xuecui; 1 Bungtown Road, Cold Spring Harbor, NY 11724 (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-MODULATED RNASE P CLEAVAGE Aptamer Target gene coding seq. Effector region 3' UTR Leader sequence AAAAAAAAAA Acceptor stem D stem loop T stem loop U Anticodon stem loop o Fig. la (57) : The present disclosure provides polynucleotide constructs for the modulation of target gene expression by aptamer-me- ---- diated ribonuclease cleavage of the target gene RNA and methods of using the constructs to modulate gene expression in response to the presence or absence of a ligand that binds the aptamer. The polynucleotide constructs contains a ribonuclease substrate sequence 0 (e.g., an RNase P substrate) and a riboswitch comprising an effector region and an aptamer such that when the aptamer binds a ligand, target gene expression occurs. C [Continued on next page] WO 2018/161053 Al MIDEDIMOMOIDEIREEMOMOHEMMIIMEIMOVOIS 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))