Patent CA-3088547-A1 (CA)
Inventor: OLSON ERIC N
IPC: C12N5/074
Filed: 2019-01-31
Abstract: CA 03088547 2020-07-14 (12) INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property 1 11111 1111111 11 111111 111 11111 11111111 1 1 111 1111 1111 1111 111 111111111 11111111111 111 1111 Organization International Bureau (10) International Publication Number (43) International Publication Date WO 2019/152609 Al 08 August 2019 (08.08.2019) WIPO I PCT (51) International Patent Classification: C12N 15/113 (2010.01) A61K 38/46 (2006.01) Published: C12N 5/074 (2010.01) ¨ with international search report (Art. 21(3)) ¨ before the expiration of the time limit for amending the (21) International Application Number: claims and to be republished in the event of receipt of PCT/US2019/015988 amendments (Rule 48.2(h)) ¨ with sequence listing part of description (Rule 5.2(a)) (22) International Filing Date: 31 January 2019 (31.01.2019) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/624,748 31 January 2018 (31.01.2018) US (71) Applicant: THE BOARD OF REGENTS OF THE UNIVERSITY OF TEXAS SYSTEM [US/US]; 210 West 7th Street, Austin, TX '78'701 (US). (72) Inventors: OLSON, Eric, N.; c/o UT Southwestern Med-ical Center, 5323 Harry Hines Blvd., Dallas, TX 75390 (US). LONG, Chengzu; c/o New York University School of Medicine, 522 First Avenue, Smilow '701C, New York, NY 10016 (US). = (74) Agent: HIGHLANDER, Steven, L.; Parker Highlander PLLC, 1120 S. Capital of Texas Highway, Bldg. One, Suite 200, Austin, TX 78746 (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, ER, 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, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). r=11 _____________________________________________________________________________ kin (54) Title: COMPOSITIONS AND METHODS FOR CORRECTING DYSTROPHIN MUTATIONS IN HUMAN CARDIOMY-OCYTES (57) Abstract: The disclosure provides a method for treating or preventing Duchene Muscular Dystrophy (DMD) in a subject in need 0 thereof, the method comprising administering to the subject a Cas9 nuclease or a sequence encoding a Cas9 nuclease, and a gRNA or a el sequence encoding a gRNA, wherein the gRNA targets a splice donor or splice acceptor site of the dystrophin gene. The administering C restores dystrophin expression in at least a subset of the subjects cardiomyocytes, and may at least partially or fully restore cardiac contractility.