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1. (WO2015035364) PROTEINS FOR TARGETING NEURONAL NITRIC OXIDE SYNTHASE TO MUSCLE SARCOLEMMA AND RELATED METHODS OF USE
Latest bibliographic data on file with the International Bureau   

Pub. No.:    WO/2015/035364    International Application No.:    PCT/US2014/054727
Publication Date: 12.03.2015 International Filing Date: 09.09.2014
IPC:
C12N 15/62 (2006.01)
Applicants: UNIVERSITY OF WASHINGTON THROUGH ITS CENTER FOR COMMERCIALIZATION [US/US]; 4311 11th Avenue NE, Suite 500 Seattle, Washington 98105 (US)
Inventors: FROEHNER, Stanley C.; (US).
ADAMS, Marvin; (US).
KIM, Min Jeong; (US).
LOPEZ, Daniela Rebolledo; (CL).
WHITEHEAD, Nicholas P.; (US)
Agent: FITZGERALD, Mark J.; (US)
Priority Data:
61/875,477 09.09.2013 US
Title (EN) PROTEINS FOR TARGETING NEURONAL NITRIC OXIDE SYNTHASE TO MUSCLE SARCOLEMMA AND RELATED METHODS OF USE
(FR) PROTÉINES POUR LE CIBLAGE D'OXYDE NITRIQUE SYNTHASE NEURONALE AU SARCOLEMME MUSCULAIRE ET PROCÉDÉS D'UTILISATION ASSOCIÉS
Abstract: front page image
(EN)It is demonstrated herein that nNOS-µ is an important modulator of muscle function, and that the loss of nNOS-µ contributes to pathogenesis in neuromuscular diseases, including DMD. Furthermore, the impact of the restoration of cytoplasmic and sarcolemma-localized nNOS-µ on mdx skeletal muscle pathology and function is demonstrated. Accordingly, provided herein are compositions, including therapeutic and pharmaceutical compositions, comprising nNOS-µ fusion proteins and derivatives thereof, and methods of treating neuromuscular disorders using such nNOS-µ fusion proteins.
(FR)Il est démontré par les présentes que nNOS-µ est un modulateur important de la fonction musculaire et que la perte de nNOS-µ contribue à la pathogenèse dans des maladies neuromusculaires comprenant DMD. De plus, l'effet de la restauration de nNOS-µ cytoplasmique et localisé dans le sarcolemme sur la pathologie et la fonction du muscle squelettique liées à mdx est démontré. Ainsi, la présente invention concerne des compositions, comprenant des compositions thérapeutiques et pharmaceutiques, comportant des protéines hybrides nNOS-µ et des dérivés de celles-ci, et des méthodes de traitement de troubles neuromusculaires à l'aide de telles protéines hybrides de nNOS-µ.
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)