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1. (WO2010084136) A DOMINANT NEGATIVE MUTANTS OF SAM68 FOR USE IN THE TREATMENT OF SPINAL MUSCULAR ATROPHY (SMA)
Latest bibliographic data on file with the International Bureau

Pub. No.: WO/2010/084136 International Application No.: PCT/EP2010/050650
Publication Date: 29.07.2010 International Filing Date: 20.01.2010
IPC:
A61K 48/00 (2006.01) ,C07K 14/47 (2006.01)
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48
Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
K
PEPTIDES
14
Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435
from animals; from humans
46
from vertebrates
47
from mammals
Applicants:
FONDAZIONE SANTA LUCIA [IT/IT]; Via Ardeatina, 306 I-00179 Roma, IT (AllExceptUS)
PARONETTO, Maria Paola [IT/IT]; IT (UsOnly)
PEDROTTI, Simona [IT/IT]; IT (UsOnly)
Inventors:
PARONETTO, Maria Paola; IT
PEDROTTI, Simona; IT
Agent:
JORIO, Paolo; Studio Torta S.r.l. Via Viotti, 9 I-10121 Torino, IT
Priority Data:
RM2009A 00002121.01.2009IT
Title (EN) A DOMINANT NEGATIVE MUTANTS OF SAM68 FOR USE IN THE TREATMENT OF SPINAL MUSCULAR ATROPHY (SMA)
(FR) MUTANTS NÉGATIFS DOMINANTS DE SAM68 DESTINÉS À ÊTRE UTILISÉS POUR LE TRAITEMENT D'UNE ATROPHIE MUSCULAIRE SPINALE (SMA)
Abstract:
(EN) The present invention relates to the use of dominant negative mutants of Sam68 for the manufacture of a medicament for the treatment of spinal muscular atrophy, to nucleic acids coding for such mutants and to vectors and methods related thereto.
(FR) La présente invention porte sur l'utilisation de mutants négatifs dominants de SAM68 pour la fabrication d'un médicament destiné au traitement d'une atrophie musculaire spinale, sur des acides nucléiques codants pour de tels mutants et sur des facteurs et des procédés apparentés.
Designated States: AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, 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, IS, JP, KE, KG, KM, KN, KP, KR, KZ, LA, LC, LK, LR, LS, LT, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PE, PG, PH, PL, PT, RO, RS, RU, 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, 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, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, SE, SI, SK, SM, 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)
Also published as:
EP2384202US20120071415