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Machine translation
1. (WO2018085688) GENE TRANSFER COMPOSITIONS, METHODS AND USES FOR TREATING NEURODEGENERATIVE DISEASES
Latest bibliographic data on file with the International Bureau    Submit observation

Pub. No.: WO/2018/085688 International Application No.: PCT/US2017/059986
Publication Date: 11.05.2018 International Filing Date: 03.11.2017
IPC:
A61K 31/343 (2006.01) ,A61K 31/5377 (2006.01) ,A61K 35/76 (2015.01)
Applicants: THE CHILDREN'S HOSPITAL OF PHILADELPHIA[US/US]; 3401 Civic Center Boulevard Philadelphia, Pennsylvania 19104, US
Inventors: DAVIDSON, Beverly L.; US
CHEN, Yong Hong; US
TECEDOR, Luis; US
Agent: BEDGOOD, Robert M.; US
Priority Data:
62/418,03304.11.2016US
Title (EN) GENE TRANSFER COMPOSITIONS, METHODS AND USES FOR TREATING NEURODEGENERATIVE DISEASES
(FR) COMPOSITIONS DE TRANSFERT DE GÈNE, MÉTHODES ET UTILISATIONS POUR LE TRAITEMENT DE MALADIES NEURODÉGÉNÉRATIVES
Abstract: front page image
(EN) Provided are methods of treating a lysosomal storage disorder in a mammal which method includes administering AAV particles encoding a polypeptide to the central nervous system of the mammal. AAV particles may be delivered by direct injection into the brain, spinal cord, cerebral spinal fluid or a portion thereof for expression.
(FR) L'invention concerne des méthodes de traitement d'un trouble de stockage lysosomal chez un mammifère, laquelle méthode comprend l'administration, dans le système nerveux central du mammifère, de particules d'AAV codant pour un polypeptide. Les particules d'AAV peuvent être administrées par injection directe dans le cerveau, la moelle épinière, le liquide céphalo-rachidien ou une partie de ceux-ci en vue de leur expression.
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 (ARIPO) (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Office (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (EPO) (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)