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1. (WO2010083338) MODULATION OF PRE-MRNA USING SPLICE MODULATING OLIGONUCLEOTIDES AS THERAPEUTIC AGENTS IN THE TREATMENT OF DISEASE
Latest bibliographic data on file with the International Bureau

Pub. No.: WO/2010/083338 International Application No.: PCT/US2010/021078
Publication Date: 22.07.2010 International Filing Date: 14.01.2010
IPC:
A61K 48/00 (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
Applicants:
PHILADELPHIA HEALTH AND EDUCATION CORPORATION [US/US]; D/b/a Drexel Universtiy College Of Medicine 245 N. 15th Street Philadelphia, Pennsylvania 19102, US (AllExceptUS)
LUTZ, Gordon, J. [US/US]; US (UsOnly)
TALLENT, Melanie K. [US/US]; US (UsOnly)
LYKENS, Nicole Michele [US/US]; US (UsOnly)
Inventors:
LUTZ, Gordon, J.; US
TALLENT, Melanie K.; US
LYKENS, Nicole Michele; US
Agent:
HAAS, Dennis L.; Drinker Biddle & Reath LLP One Logan Square, 18th and Cherry Streets Philadelphia, PA 19103, US
Priority Data:
61/144,54314.01.2009US
Title (EN) MODULATION OF PRE-MRNA USING SPLICE MODULATING OLIGONUCLEOTIDES AS THERAPEUTIC AGENTS IN THE TREATMENT OF DISEASE
(FR) MODULATION DE PRE-ARNM AU MOYEN D'OLIGONUCLEOTIDES DE MODULATION D'EPISSAGE SERVANT D'AGENTS THERAPEUTIQUES DANS LE TRAITEMENT D'UNE MALADIE
Abstract:
(EN) The present invention encompasses a class of compounds known as splice modulating oligonucleotides (SMOs) that modulate pre-mRNA splicing, thereby affecting expression and functionality of a specific protein in a cell. The present invention further provides compositions and methods for modulating pre-mRNA splicing using a SMO of the invention to abrogate disease-causing mutations in a protein. Accordingly, the present invention provides compositions and methods of treating a subject at risk of, susceptible to, or having a disease, disorder, or condition associated with aberrant or unwanted target pre-mRNA expression or activity.
(FR) L'invention concerne une catégorie de composés appelés oligonucléotides de modulation d'épissage (SMO) modulant l'épissage de pré-ARNm, ce qui modifie l'expression et la fonctionnalité d'une protéine spécifique dans une cellule. L'invention concerne également des compositions et des méthodes de modulation d'épissage de pré-ARNm au moyen d'un SMO selon l'invention, pour supprimer les mutations pathogènes dans une protéine. Ainsi, la présente invention concerne des compositions et des méthodes permettant de traiter un patient à risque ou susceptible de développer une maladie, affection ou trouble associé à l'expression ou l'activité aberrante ou indésirable de pré-ARNm cibles.
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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:
EP2381965US20120316223CA2750379AU2010204639