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1. (WO2016179038) ADENO-ASSOCIATED VIRUS-MEDIATED CRISPR-Cas9 TREATMENT OF OCULAR DISEASE
Latest bibliographic data on file with the International Bureau   

Pub. No.:    WO/2016/179038    International Application No.:    PCT/US2016/030273
Publication Date: 10.11.2016 International Filing Date: 29.04.2016
IPC:
A61K 48/00 (2006.01), A61K 39/235 (2006.01), A61P 27/02 (2006.01)
Applicants: SPARK THERAPEUTICS, INC. [US/US]; 3737 Market Street, Suite 1300 Philadelphia, Pennsylvania 19104 (US).
BUCHLIS, George [US/US]; (US).
ANGUELA, Xavier [ES/US]; (US).
HIGH, Katherine A. [US/US]; (US)
Inventors: BUCHLIS, George; (US).
ANGUELA, Xavier; (US).
HIGH, Katherine A.; (US)
Agent: BEDGOOD, Robert M.; (US)
Priority Data:
62/156,025 01.05.2015 US
Title (EN) ADENO-ASSOCIATED VIRUS-MEDIATED CRISPR-Cas9 TREATMENT OF OCULAR DISEASE
(FR) TRAITEMENT D'UNE MALADIE OCULAIRE PAR UN SYSTÈME CRISPR-CAS9 MÉDIÉ PAR UN VIRUS ADÉNO-ASSOCIÉ
Abstract: front page image
(EN)Disclosed herein are compositions and methods of treating and/or correcting ocular disease in a subject, such as a mammal (e.g., human) eye using an Adeno- associated virus (AAV) system. The AAV system employs a nucleic acid encoding a CRISPR-Cas9 system for targeted gene disruption or correction.
(FR)L'invention concerne des compositions et des méthodes de traitement et/ou de correction d'une maladie oculaire chez un sujet, tel qu'un oeil de mammifère (par exemple, humain) à l'aide d'un système de virus adéno-associé (AAV). Le système AVV utilise un acide nucléique codant pour un système CRISPR-Cas9 pour la disruption ou la correction génique ciblée.
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)