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1. (WO2014121287) SELECTIVE ANTISENSE COMPOUNDS AND USES THEREOF
Latest bibliographic data on file with the International Bureau

Pub. No.: WO/2014/121287 International Application No.: PCT/US2014/014722
Publication Date: 07.08.2014 International Filing Date: 04.02.2014
IPC:
C12N 15/11 (2006.01) ,A61K 48/00 (2006.01) ,C07H 21/00 (2006.01)
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15
Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09
Recombinant DNA-technology
11
DNA or RNA fragments; Modified forms thereof
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
H
SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
21
Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
Applicants:
ISIS PHARMACEUTICALS, INC. [US/US]; 2855 Gazelle Court Carlsbad, CA 92010, US
Inventors:
OESTERGAARD, Michael; US
SETH, Punit, P.; US
SWAYZE, Eric, E.; US
Agent:
SCARR, Rebecca; McNeill Baur PLLC 125 Cambridge Park Dr, Suite 301 Cambridge, MA 02140, US
Priority Data:
61/760,61004.02.2013US
Title (EN) SELECTIVE ANTISENSE COMPOUNDS AND USES THEREOF
(FR) COMPOSÉS ANTISENS SÉLECTIFS ET LEURS UTILISATIONS
Abstract:
(EN) The present disclosure provides oligomeric compounds. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount activity or expression of the target nucleic acid in a cell. In certain embodiments, certain oligomeric compounds selectively reduce the expression of a target nucleic acid transcript relative to a non-target nucleic acid transcript.
(FR) Cette invention concerne des composés oligomères. Certains de ces composés oligomères sont utiles pour s'hybrider à un acide nucléique complémentaire, comprenant, entre autres, un acide nucléique dans une cellule. Dans certains modes de réalisation, l'hybridation induit la modulation quantitative de l'activité ou de l'expression de l'acide nucléique cible dans une cellule. Dans d'autres modes de réalisation, certains composés oligomères réduisent sélectivement l'expression d'un transcrit d'acide nucléique cible par rapport au transcrit d'un acide nucléique non ciblé.
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, LT, 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 (ARIPO) (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, 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)