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1. (WO2009139630) METHOD FOR EFFICIENT EXON (44) SKIPPING IN DUCHENNE MUSCULAR DYSTROPHY AND ASSOCIATED MEANS
Latest bibliographic data on file with the International Bureau   

Pub. No.:    WO/2009/139630    International Application No.:    PCT/NL2009/050258
Publication Date: 19.11.2009 International Filing Date: 14.05.2009
IPC:
C12N 15/11 (2006.01), A61K 31/7088 (2006.01), C12N 15/86 (2006.01)
Applicants: PROSENSA TECHNOLOGIES B.V. [NL/NL]; Wassenaarseweg 72 NL-2333 AL Leiden (NL) (For All Designated States Except US).
ACADEMISCH ZIEKENHUIS LEIDEN [NL/NL]; Albinusdreef 2 NL-2333 ZA Leiden (NL) (For All Designated States Except US).
PLATENBURG, Gerardus Johannes [NL/NL]; (NL) (For US Only).
DE KIMPE, Josephus Johannes [NL/NL]; (NL) (For US Only).
VAN DEUTEKOM, Judith Christina Theodora [NL/NL]; (NL) (For US Only).
VAN OMMEN, Garrit-Jan Boudewijn [NL/NL]; (NL) (For US Only).
AARTSMA-RUS, Annemieke [NL/NL]; (NL) (For US Only)
Inventors: PLATENBURG, Gerardus Johannes; (NL).
DE KIMPE, Josephus Johannes; (NL).
VAN DEUTEKOM, Judith Christina Theodora; (NL).
VAN OMMEN, Garrit-Jan Boudewijn; (NL).
AARTSMA-RUS, Annemieke; (NL)
Agent: Nederlandsch Octrooibureau; Att: Mrs C. Pallard P.O. Box 29720 NL-2502 LS The Hague (NL)
Priority Data:
08156193.8 14.05.2008 EP
61/128,010 15.05.2008 US
Title (EN) METHOD FOR EFFICIENT EXON (44) SKIPPING IN DUCHENNE MUSCULAR DYSTROPHY AND ASSOCIATED MEANS
(FR) PROCÉDÉ EFFICACE D'OMISSION D'EXON (44) DANS LA DYSTROPHIE MUSCULAIRE DE DUCHENNE ET MOYEN ASSOCIÉ
Abstract: front page image
(EN)The invention relates to a nucleic acid molecule that binds and/or is complementary to the nucleotide molecule having sequence 5'-GUGGCUAACAGAAGCU (SEQ ID NO 1) and to its use in a method for inducing skipping of exon 44 of the DMD gene in a DMD patient.
(FR)L'invention concerne une molécule d'acide nucléique qui se lie et/ou est complémentaire de la molécule de nucléotide ayant la séquence 5'-GUGGCUAACAGAAGCU (SEQ ID NO 1) et son utilisation dans un procédé d'induction de l'omission de l'exon 44 du gène DMD chez un patient DMD.
Designated States: AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BR, BW, BY, BZ, CA, CH, 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, PG, PH, PL, PT, RO, RS, RU, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW.
African Regional Intellectual Property Organization (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, 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)