Patent SG-11201809096V-A (SG)
Title: Compositions and methods for enhanced gene expression of pklr
Inventor: SEGOVIA JOSE C
IPC: C12N15/79
Filed: 2017-04-20
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 26 October 2017 (26.10.2017) WIPO I PCT omit VIII Hot 0 OH moimmollii °nom mil (10) International Publication Number WO 2017/184903 Al (51) International Patent Classification: A61K 48/00 (2006.01) Cl 2N 15/54 (2006.01) Cl 2N 15/79 (2006.01) (21) International Application Number: PCT/US2017/028695 (22) International Filing Date: 20 April 2017 (20.04.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/325,397 20 April 2016 (20.04.2016) US (71) Applicants: CENTRO DE INVESTIGACIONES EN- ERGETICAS, MEDIOAMBIENTALES Y TEC- NOLOGICAS [ES/ES]; 8-2 Address: Avda. de la Com- plutense, 40, 28040 Madrid (ES). FUNDACION INSTI- TUTO DE INVESTIGACION SANITARIA FUN- DACION JIMENEZ DIAZ [ES/ES]; 8-3 Address: Avda. de los Reyes Catolicos, 2, 28040 Madrid (ES). CENTRO DE INVESTIGACION BIOMEDICA EN RED [ES/ES]; 8-1 Address: Monforte de Lemos, 5, 28029 Madrid (ES). (72) Inventors; and (71) Applicants: SEGOVIA, Jose C. [ES/ES]; Avda. de la Complutense, 40, 28040 Madrid (ES). GOMEZ, Maria G. [ /GB]; London (GB). NAVARRO, Susana [ES/ES]; Av- da. de la Complutense, 40, 28040 Madrid (ES). MEZA, Nestor [ /VE]; San Cristobal (VE). BUEREN, Juan [ES/ES]; Avda. de la Complutense, 40, 28040 Madrid (ES). BRAVO, Maria G. [ /ES]; Madrid (ES). (74) Agent: LAHERTY, Carol D. et al.; Cooley LLP, 1299 Pennsylvania Avenue, NW, Suite 700, Washington, District of Columbia 20004 (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). Published: 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)) (54) Title: COMPOSITIONS AND METHODS FOR ENHANCED GENE EXPRESSION OF PKLR 5' LTR 3' LTR 1-1 O co N FIG. 21 \" (57) : The present disclosure provides polynucleotide cassettes, expression vectors and methods for the expression of a gene in mammalian cells to provide gene therapy for pyruvate kinase deficiency.