WIPO logo
Mobile | Deutsch | Español | Français | 日本語 | 한국어 | Português | Русский | 中文 | العربية |
PATENTSCOPE

Search International and National Patent Collections
World Intellectual Property Organization
Search
 
Browse
 
Translate
 
Options
 
News
 
Login
 
Help
 
Machine translation
1. (WO2015120450) ANTISENSE OLIGONUCLEOTIDES FOR TREATMENT OF SPINAL MUSCULAR ATROPHY
Latest bibliographic data on file with the International Bureau   

Pub. No.:    WO/2015/120450    International Application No.:    PCT/US2015/015212
Publication Date: 13.08.2015 International Filing Date: 10.02.2015
IPC:
C12N 15/11 (2006.01), C07H 21/02 (2006.01)
Applicants: OHIO STATE INNOVATION FOUNDATION [US/US]; 1524 North High Street Columbus, OH 43201 (US)
Inventors: BURGHES, Arthur; (US).
MCGOVERN, Vicki; (US).
PRIOR, Thomas; (US)
Agent: JEFFERIES, David E.; (US)
Priority Data:
61/937,828 10.02.2014 US
Title (EN) ANTISENSE OLIGONUCLEOTIDES FOR TREATMENT OF SPINAL MUSCULAR ATROPHY
(FR) OLIGONUCLÉOTIDES ANTISENS DANS LE TRAITEMENT DE L'AMYOTROPHIE SPINALE
Abstract: front page image
(EN)Various aspects of the present invention are directed to compounds targeted to various regions of the survival motor neuron 2 (SMN2) gene. Such compounds may be used to increase incorporation of exon 7 in processed transcripts of SMN2. Such compounds may therefore be useful in increasing the amount of full-length SMN protein produced by the SMN2 gene. As such, these compounds may provide a therapeutic approach for treatment of spinal muscular atrophy (SMA).
(FR)Divers aspects de la présente invention concernent des composés ciblant diverses régions du gène de survie des neurones moteurs 2 (SMN2). De tels composés peuvent être utilisés pour augmenter l'incorporation de l'exon 7 dans des produits de transcription traités de SMN2. De tels composés peuvent par conséquent être utiles dans l'augmentation de la quantité de protéine SMN de longueur complète produite par le gène SMN2. En tant que tels, ces composés peuvent fournir une approche thérapeutique du traitement de l'amyotrophie spinale (SMA).
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, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KN, KP, KR, 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)