Patent SG-11201803971U-A (SG)
Title: Methods of treating muscular dystrophy
Inventor: RODINO-KLAPAC LOUISE
IPC: C07K14/435
Filed: 2016-11-11
Abstract: INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property - Organization International Bureau (43) International Publication Date .... ..sr ,„„..) 18 May 2017 (18.05.2017) WIPO I PCT (10) WO International Publication Number 111111111111311111111111111111111111111111111111111111111111111111111311111111111111111 2017/083776 Al (51) International Patent Classification: AO, AT, AU, AZ, BA, A61K 48/00 (2006.01) A61P 39/06 (2006.01) BZ, CA, CH, A61P 21/00 (2006.01) CO7K 14/435 (2006.01) DO, DZ, EC, HN, HR, HU, (21) International Application Number: KW, KZ, LA, PCT/US2016/061703 MG, MK, MN, MW, MX, (22) International Filing Date: OM, PA, PE, PG, PH, PL, 11 November 2016 (11.11.2016) SC, SD, SE, SG, SK, SL, TN, TR, TT, (25) Filing Language: English ZW. (26) Publication Language: English (84) Designated (30) Priority Data: kind of regional 62/254,539 12 November 2015 (12.11.2015) US GM, KE, LR, 62/419,793 9 November 2016 (09.11.2016) US TZ, UG, ZM, TJ, TM), European (71) Applicant: RESEARCH INSTITUTE AT NATION- DK, EE, ES, FI, FR, GB, CL, CN, CO, EE, EG, ES, ID, IL, IN, LC, LK, TZ, UA, States (unless protection LS, MW, ZW), Eurasian (AL, BB, BG, BH, BN, BR, BW, BY, CR, CU, CZ, DE, DJ, DK, DM, FI, GB, GD, GE, GH, GM, GT, IR, IS, JP, KE, KG, KN, KP, KR, LR, LS, LU, LY, MA, MD, ME, MY, MZ, NA, NG, NI, NO, NZ, PT, QA, RO, RS, RU, RW, SA, SM, ST, SV, SY, TH, TJ, TM, UG, US, UZ, VC, VN, ZA, ZM, otherwise indicated, for every available): ARIPO (BW, GH, MZ, NA, RW, SD, SL, ST, SZ, (AM, AZ, BY, KG, KZ, RU, AT, BE, BG, CH, CY, CZ, DE, GR, HR, HU, IE, IS, IT, LT, LU, WIDE CHILDREN'S HOSPITAL [US/US]; 700 Chil- LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, dren's Drive, Room W 172, Columbus, OH 43205 (US). SM, TR), OAPI GW, KM, (72) Inventor: RODINO-KLAPAC, Louise; 4912 Bixby Ridge Drive, E. Groveport, OH 43125 (US). Published: (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, ML, MR, NE, SN, TD, TG). (74) Agent: SINTICH, Sharon, M.; Marshall, Gerstein & — with international search report (Art. 21(3)) Borun LLP, 233 S. Wacker Drive, 6300 Willis Tower, with sequence listing part of description (Rule 5.2(a)) Chicago, IL 60606-6357 (US). (81) Designated States (unless otherwise indicated, for every kind of national available): AE, AG, AL, AM, protection (54) Title: METHODS OF TREATING MUSCULAR DYSTROPHY Figure 1 a . . . C . 0 2 Ex 8 on Exon 9 o Ca o -0 t . \"E a 5' arm 3' arm SV40 glox IRE lox FRT lox FRT polyA ia a ' - S P P P e3 0 at 2 . 71 , .... 2 8 , 3 — E tn. Exon 8 A cx 0 -.0 Exogenous LacZ NEOR (a arm intron cassette 3' arm b c d WT +1- -1- WT 11 Gapdh e10-14 e17-20 c Ano5 ex4-5 Ano5 ex16-17 .4t g 120% 120% ovrt oAno5-/- owt o Ano5-/- -±- :(c) 100% Q- R 8 0 °/o 0 cc ' 0 C my. I I r 00% 80°. r- . I / IN Ano5-/- -D 60% '' 60\" ' . „ I Ir...4 tis i lL''\" Gapdh e10-14 e17-20 1 11 I NE I. 0\" 4 0 : M c“ 20V ; 14 n 41 n A 2 0c ; I 14 4 Ge ' EL' \" Quad Gastroc. T.A.' Quad Gastroc TA o Genomic RT PCR qRT PCR IN 1-1 (57) : The invention provides for AAV vectors expressing the ANO5 gene and antioxidant therapy as methods of inducing 0 muscle regeneration and a method of treating muscular dystrophy. el 0