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1. (WO2004098648) GENE THERAPY FOR NEUROMETABOLIC DISORDERS
Latest bibliographic data on file with the International Bureau   

Pub. No.:    WO/2004/098648    International Application No.:    PCT/US2004/013685
Publication Date: 18.11.2004 International Filing Date: 30.04.2004
IPC:
A61K 38/46 (2006.01), A61K 48/00 (2006.01), C12N 15/864 (2006.01)
Applicants: PASSINI, Marco, A. [US/US]; (US) (For US Only).
GENZYME CORPORATION [US/US]; 15 Pleasant Street Connector, Framingham, MA 01701-9322 (US) (For All Designated States Except US).
STEWART, Greg [US/US]; (US) (For US Only)
Inventors: PASSINI, Marco, A.; (US).
STEWART, Greg; (US)
Agent: GARRETT, Arthur, S.; Finnegan, Henderson, Farabow, Garrett & Dunner, L.L.P., 1300 I Street, N.W., Washington, DC 20005-3315 (US)
Priority Data:
60/467,195 01.05.2003 US
60/475,726 04.06.2003 US
Title (EN) GENE THERAPY FOR NEUROMETABOLIC DISORDERS
(FR) THERAPIE GENIQUE POUR TROUBLES NEUROMETABOLIQUES
Abstract: front page image
(EN)The disclosure pertains to methods for treating neurometabolic disorder including lysosomal storage diseases that affect the central nervous system, e.g., Niemann-Pick A disease. The disclosed methods involve contacting an axonal ending of a neuron with a composition containing high titer AAV carrying a therapeutic transgene so that the AAV vector is axonally transported in a retrograde fashion and transgene product is expressed distally to the administration site.
(FR)L'invention concerne des méthodes de traitement de troubles neurométaboliques, notamment de maladies lysosomales qui affectent le système nerveux central, par exemple, la maladie de Niemann-Pick A. Les méthodes selon l'invention consistent à mettre en contact une terminaison axonale d'un neurone avec une composition contenant un AAV à titre élevé portant un transgène thérapeutique de sorte que le vecteur AAV soit transporté par voie axonale de manière rétrograde et que le produit transgénique soit exprimé de manière distale par rapport au site d'administration.
Designated States: AE, AG, AL, AM, AT, AU, AZ, BA, BB, BG, BR, BW, BY, BZ, CA, CH, CN, CO, CR, CU, CZ, DE, DK, DM, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, HR, HU, ID, IL, IN, IS, JP, KE, KG, KP, KR, KZ, LC, LK, LR, LS, LT, LU, LV, MA, MD, MG, MK, MN, MW, MX, MZ, NA, NI, NO, NZ, OM, PG, PH, PL, PT, RO, RU, SC, SD, SE, SG, SK, SL, SY, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, YU, ZA, ZM, ZW.
African Regional Intellectual Property Organization (BW, GH, GM, KE, LS, MW, MZ, NA, SD, SL, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Organization (AM, AZ, BY, KG, KZ, MD, RU, TJ, TM)
European Patent Office (AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HU, IE, IT, LU, MC, NL, PL, PT, RO, SE, SI, SK, TR)
African Intellectual Property Organization (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, ML, MR, NE, SN, TD, TG).
Publication Language: English (EN)
Filing Language: English (EN)