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1. (WO2016100575) GENE THERAPY FOR JUVENILE BATTEN DISEASE
Latest bibliographic data on file with the International Bureau   

Pub. No.:    WO/2016/100575    International Application No.:    PCT/US2015/066206
Publication Date: 23.06.2016 International Filing Date: 16.12.2015
IPC:
A61K 48/00 (2006.01), C12N 15/861 (2006.01)
Applicants: BOARD OF REGENTS OF THE UNIVERSITY OF NEBRASKA [US/US]; Varner Hall, 3835 Holdredge Street Lincoln, Nebraska 68583-0745 (US).
OHIO STATE INNOVATION FOUNDATION [US/US]; 1524 North High Street Columbus, Ohio 43201 (US)
Inventors: KIELIAN, Tammy; (US).
FOUST, Kevin; (US)
Agent: KONSKI, Antoinette F.; (US)
Priority Data:
62/092,501 16.12.2014 US
62/146,793 13.04.2015 US
Title (EN) GENE THERAPY FOR JUVENILE BATTEN DISEASE
(FR) THÉRAPIE GÉNIQUE POUR LA MALADIE DE STEINERT JUVÉNILE
Abstract: front page image
(EN)Compositions and methods for the treatment of Juvenile Neuronal Ceroid Lipofuscinosis (JNCL), also known as Juvenile Batten Disease, are provided herein. In certain embodiments the compositions include but are not limited to adeno-associated viral (AAV) constructs, including self-complementary adeno-associated viral (sc-AAV) constructs, that express the human gene CLN3 (or a CLN3 cDNA).
(FR)La présente invention concerne des compositions et des procédés pour le traitement de la maladie de Spielmeyer-Voght juvénile, également connue sous le nom de la maladie de Steinert juvénile. Dans certains modes de réalisation, les compositions comprennent non exclusivement des constructions de virus adéno-associées (AAV), y compris des constructions de virus adéno-associées auto-complémentaires (sc-AAV), qui expriment le gène humain CLN3 (ou un ADNc CLN3).
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)