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1. (WO2013123217) SMALL MOLECULES THAT ENHANCE THE ACTIVITY OF OLIGONUCLEOTIDES
Latest bibliographic data on file with the International Bureau   

Pub. No.: WO/2013/123217 International Application No.: PCT/US2013/026176
Publication Date: 22.08.2013 International Filing Date: 14.02.2013
IPC:
C12N 5/10 (2006.01) ,C12N 5/071 (2010.01) ,C12N 15/113 (2010.01) ,C07D 243/00 (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
5
Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
10
Cells modified by introduction of foreign genetic material, e.g. virus-transformed cells
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
5
Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
07
Animal cells or tissues
071
Vertebrate cells or tissues, e.g. human cells or tissues
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
113
Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
D
HETEROCYCLIC COMPOUNDS
243
Heterocyclic compounds containing seven-membered rings having two nitrogen atoms as the only ring hetero atoms
Applicants:
THE UNIVERSITY OF NORTH CAROLINA AT CHAPEL HILL [US/US]; 308 Bynum Hall Campus 4105 Chapel Hill, North Carolina 27599-4105, US
COMMISSARIAT À L'ENERGIE ATOMIQUE ET AUX ENERGIES ALTERNATIVES [FR/FR]; 25, rue Leblanc Bâtiment "le Ponant D" F-75015 Paris, FR
Inventors:
JULIANO, Rudolph; US
MING, Xin; US
FISHER, Michael H.; US
GILLET, Daniel; FR
BARBIER, Julien; FR
Agent:
MYERS BIGEL SIBLEY & SAJOVEC, P.A.; P.O. Box 37428 Raleigh, North Carolina 27627, US
Priority Data:
61/599,67316.02.2012US
Title (EN) SMALL MOLECULES THAT ENHANCE THE ACTIVITY OF OLIGONUCLEOTIDES
(FR) PETITES MOLÉCULES RENFORÇANT L'ACTIVITÉ DES OLIGONUCLÉOTIDES
Abstract:
(EN) A method of administering an oligonucleotide of interest into a cell (e.g., a mammalian cell) is carried out by concurrently administering an intracellular trafficking route inhibitor to the cell in an amount effective to increase the delivery and/or increase the activity of said oligonucleotide in said cell. In some embodiments, the intracellular trafficking inhibitor is an intracellular retrograde transport inhibitor (retro compound) such as an imine derivative or a benzodiazepine derivative. Compositions useful for carrying out such methods are also described.
(FR) La présente invention concerne un procédé d'administration d'un oligonucléotide d'intérêt dans une cellule (par exemple, une cellule de mammifère), un inhibiteur de voie de trafic intracellulaire étant administré en même temps à la cellule en une quantité efficace pour améliorer l'administration et/ou renforcer l'activité dudit oligonucléotide dans ladite cellule. Dans certains modes de réalisation, l'inhibiteur de trafic intracellulaire est un inhibiteur de transport rétrograde intracellulaire (composé rétro), tel qu'un dérivé d'imine ou un dérivé de benzodiazépine. L'invention concerne également des compositions permettant de mettre en œuvre ce procédé.
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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, 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, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, 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 Office (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (EPO) (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)