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1. (WO2011154427) MODIFIED snRNAs FOR USE IN THERAPY
Latest bibliographic data on file with the International Bureau   

Pub. No.:    WO/2011/154427    International Application No.:    PCT/EP2011/059435
Publication Date: 15.12.2011 International Filing Date: 08.06.2011
IPC:
C12N 15/11 (2006.01), C12N 15/113 (2010.01), A61K 31/7088 (2006.01)
Applicants: AMSTERDAM MOLECULAR THERAPEUTICS (AMT) IP B.V. [NL/NL]; Meibergdreef 61 NL-1105 BA Amsterdam (NL) (For All Designated States Except US).
BOZZONI, Irene [IT/IT]; (IT) (For US Only).
DE ANGELIS, Fernanda [IT/IT]; (IT) (For US Only).
INCITTI, Tania [IT/IT]; (IT) (For US Only).
MARTONE, Julie [IT/IT]; (IT) (For US Only).
CAZZELLA, Valentina [IT/IT]; (IT) (For US Only).
PINNARO', Chiara [IT/IT]; (IT) (For US Only)
Inventors: BOZZONI, Irene; (IT).
DE ANGELIS, Fernanda; (IT).
INCITTI, Tania; (IT).
MARTONE, Julie; (IT).
CAZZELLA, Valentina; (IT).
PINNARO', Chiara; (IT)
Agent: CAPASSO, Olga; c/o De Simone and Partners S.p.A. Via Vincenzo Bellini, 20 I-00198 Roma (IT)
Priority Data:
RM2010A000310 08.06.2010 IT
Title (EN) MODIFIED snRNAs FOR USE IN THERAPY
(FR) PETITS ARN NUCLÉAIRES À USAGE THÉRAPEUTIQUE
Abstract: front page image
(EN)The invention relates to a nucleic acid encoding a small nuclear RNA (snRNA), which snRNA is modified so that it contains sequence capable of hybridizing with the 5' and/or 3' splice site junctions and at least one exonic splicing enhancer of an exon of a pre-mRNA, so that the exon sequence is skipped during the splicing process that converts the pre-mRNA in the mature mRNA. The nucleic acid, the modified snRNA and vectors incorporating the nucleic acid may be used in the therapy, in particular in the treatment of muscular dystrophies.
(FR)L'invention concerne un acide nucléique codant un petit ARN nucléaire (snRNA), lequel snRNA est modifié de façon à contenir une séquence susceptible de s'hybrider avec les jonctions de sites d'épissage en 5' et/ou en 3' et au moins un amplificateur d'épissage exonique d'un EXON d'un pré-ARNm, ainsi, la séquence exon est ignorée durant le processus d'épissage qui transforme le pré-ARNm en ARNm mature. L'acide nucléique, l'ARN nucléaire modifié et les vecteurs incorporant l'acide nucléique peuvent servir en thérapie, notamment dans le traitement de dystrophies musculaires.
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 (BW, GH, GM, KE, LR, 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 (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, ML, MR, NE, SN, TD, TG).
Publication Language: English (EN)
Filing Language: English (EN)