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1. (WO2016061509) COMPOSITIONS AND METHODS OF TREATING MUSCULAR DYSTROPHY
Latest bibliographic data on file with the International Bureau   

Pub. No.:    WO/2016/061509    International Application No.:    PCT/US2015/056026
Publication Date: 21.04.2016 International Filing Date: 16.10.2015
IPC:
C07K 14/27 (2006.01)
Applicants: THE BROAD INSTITUTE, INC. [US/US]; 415 Main Street Cambridge, MA 02142 (US).
CHILDREN'S MEDICAL CENTER CORPORATION [US/US]; 55 Shattuck Street Boston, MA 02115 (US).
UNIVERSITY OF SAO PAULO [BR/BR]; 10 Conifer Lane Sao Paulo (BR)
Inventors: LINDBLAD-TOH, Kerstin; (US).
KUNKEL, Louis, M.; (US).
VIEIRA, Natassia, M.; (US).
ZATZ, Mayana; (BR)
Agent: EL-HAYEK, Roque; (US)
Priority Data:
62/065,559 17.10.2014 US
Title (EN) COMPOSITIONS AND METHODS OF TREATING MUSCULAR DYSTROPHY
(FR) COMPOSITIONS ET PROCÉDÉS DE TRAITEMENT DE LA DYSTROPHIE MUSCULAIRE
Abstract: front page image
(EN)Provided herein are compositions and methods of treating muscular dystrophy (MD), such as administering an effective amount of a composition that increases the expression of JAGl, a composition comprising a JAGl agonist, or a composition that promotes JAGl signaling. Also provided are methods of prognosing MD or evaluating responsiveness to treatment for MD, e.g., by measuring an expression level of JAGl, and methods of identifying a compound for the treatment of MD.
(FR)L'invention concerne des compositions et des procédés de traitement de la dystrophie musculaire (DM), par exemple par administration d'une quantité efficace d'une composition qui augmente l'expression de JAGl, d'une composition comprenant un agoniste de JAGl, ou d'une composition qui active la signalisation de JAGl. L'invention concerne également des procédés de pronostic de la DM ou d'évaluation de la sensibilité à un traitement de la DM, par exemple, par mesure d'un niveau d'expression de JAGl, et des procédés d'identification d'un composé pour le traitement de la DM.
Designated States: 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, 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, KN, KP, KR, 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.
African Regional Intellectual Property Organization (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Organization (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (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)
African Intellectual Property Organization (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG).
Publication Language: English (EN)
Filing Language: English (EN)