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1. (WO2018011572) GENE THERAPY
Latest bibliographic data on file with the International Bureau    Submit observation

Pub. No.:    WO/2018/011572    International Application No.:    PCT/GB2017/052044
Publication Date: 18.01.2018 International Filing Date: 12.07.2017
IPC:
C12N 9/14 (2006.01), C12N 15/67 (2006.01), C12N 15/68 (2006.01), C12N 15/864 (2006.01)
Applicants: THE UNIVERSITY OF MANCHESTER [GB/GB]; Oxford Road Manchester M13 9PL (GB).
PCHEJETSKY, Alexey [CA/CA]; (CA).
CENTRE HOSPITALIER UNIVERSITAIRE SAINTE-JUSTINE [CA/CA]; 3175 chemin de la Côte-Sainte-Catherine Suite 1.17.003 Québec H3T 1C5 (CA)
Inventors: PCHEJETSKY, Alexey; (CA).
BIGGER, Brian; (GB).
O'LEARY, Claire; (GB)
Agent: APPLEYARD LEES IP LLP; 15 Clare Road Halifax Yorkshire HX1 2HY (GB)
Priority Data:
1612104.8 12.07.2016 GB
1704634.3 23.03.2017 GB
Title (EN) GENE THERAPY
(FR) THÉRAPIE GÉNIQUE
Abstract: front page image
(EN)The present invention relates to compositions and adeno associated viral vectors comprising an optimised HGSNAT nucleic acid sequence of SEQ ID No. 1 or a derivative sequence having at least 77% homology thereof. Uses of such compositions and vectors are also contemplated along with kits of parts for their administration.
(FR)La présente invention concerne des compositions et des vecteurs viraux adénoassociés comprenant une séquence d'acides nucléiques HGSNAT optimisée de SEQ ID No. 1 ou une séquence dérivée ayant au moins 77 % d'homologie par rapport à celle-ci. Des utilisations de telles compositions et vecteurs sont également envisagées en même temps que des trousses de parties pour leur administration.
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, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, 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.
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)