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Machine translation
1. (WO2001036603) RECOMBINANT ADENO-ASSOCIATED VIRUS VIRIONS FOR THE TREATMENT OF LYSOSOMAL DISORDERS
Latest bibliographic data on file with the International Bureau   

Pub. No.:    WO/2001/036603    International Application No.:    PCT/US2000/031688
Publication Date: 25.05.2001 International Filing Date: 17.11.2000
Chapter 2 Demand Filed:    01.06.2001    
IPC:
A61K 48/00 (2006.01), C12N 9/24 (2006.01), C12N 15/864 (2006.01)
Applicants: AVIGEN, INC. [US/US]; 1201 Harbor Bay Parkway, #1000, Alameda, CA 94502 (US) (For All Designated States Except US).
CHILDREN'S HOSPITAL MEDICAL CENTER OF NORTHERN CALIFORNIA [US/US]; 747 Fifty Second Street, Oakland, CA 94609-1809 (US) (For All Designated States Except US).
PODSAKOFF, Gregory [US/US]; (US) (For US Only).
WATSON, Gordon [US/US]; (US) (For US Only).
COUTO, Linda, B. [US/US]; (US) (For US Only).
YANG, Bin [US/US]; (US) (For US Only)
Inventors: PODSAKOFF, Gregory; (US).
WATSON, Gordon; (US).
COUTO, Linda, B.; (US).
YANG, Bin; (US)
Agent: ROBINS, Roberta, L.; Robins & Associates, Suite 200, 90 Middlefied Road, Menlo Park, CA 94025 (US)
Priority Data:
60/166,097 17.11.1999 US
60/215,430 30.06.2000 US
Title (EN) RECOMBINANT ADENO-ASSOCIATED VIRUS VIRIONS FOR THE TREATMENT OF LYSOSOMAL DISORDERS
(FR) VIRIONS DE VIRUS ADENO-ASSOCIES RECOMBINANTS DESTINES AU TRAITEMENT DE MALADIES LYSOSOMALES
Abstract: front page image
(EN)AAV expression vectors and recombinant virions produced using these vectors, which include genes coding for enzymes defective or missing in lysosomal storage disorders, are described. These recombinant AAV virions are useful in the treatment of a variety of lysosomal storage disorders and the methods described herein provide for long-term, sustained expression of the defective or missing enzyme.
(FR)L'invention concerne des vecteurs à expression AAV (virus adéno-associé) et des virions recombinants fabriqués au moyen de ces vecteurs, contenant des gènes codant des enzymes défectueux ou manquants dans des maladies de stockage lysosomal. Ces virions AAV recombinants servent au traitement de diverses maladies de stockage lysosomal, et les procédés selon l'invention garantissent une expression soutenue à long terme de l'enzyme défectueux ou manquant.
Designated States: AE, AG, AL, AM, AT, AU, AZ, BA, BB, BG, BR, BY, BZ, CA, CH, CN, CR, CU, CZ, DE, DK, DM, DZ, EE, 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, NO, NZ, PL, PT, RO, RU, SD, SE, SG, SI, SK, SL, TJ, TM, TR, TT, TZ, UA, UG, US, UZ, VN, YU, ZA, ZW.
African Regional Intellectual Property Organization (GH, GM, KE, LS, MW, MZ, SD, SL, SZ, TZ, UG, ZW)
Eurasian Patent Organization (AM, AZ, BY, KG, KZ, MD, RU, TJ, TM)
European Patent Office (AT, BE, CH, CY, DE, DK, ES, FI, FR, GB, GR, IE, IT, LU, MC, NL, PT, SE, TR)
African Intellectual Property Organization (BF, BJ, CF, CG, CI, CM, GA, GN, GW, ML, MR, NE, SN, TD, TG).
Publication Language: English (EN)
Filing Language: English (EN)