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1. (WO2006081546) INHIBITOR NUCLEIC ACIDS
Latest bibliographic data on file with the International Bureau   

Pub. No.: WO/2006/081546 International Application No.: PCT/US2006/003193
Publication Date: 03.08.2006 International Filing Date: 26.01.2006
IPC:
C12N 15/11 (2006.01) ,C07H 21/02 (2006.01) ,A61K 31/713 (2006.01) ,A61K 31/7125 (2006.01) ,A61K 48/00 (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
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
02
with ribosyl as saccharide radical
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31
Medicinal preparations containing organic active ingredients
70
Carbohydrates; Sugars; Derivatives thereof
7088
Compounds having three or more nucleosides or nucleotides
713
Double-stranded nucleic acids or oligonucleotides
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31
Medicinal preparations containing organic active ingredients
70
Carbohydrates; Sugars; Derivatives thereof
7088
Compounds having three or more nucleosides or nucleotides
7125
Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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:
DAVIS, Mark, E. [US/US]; US (UsOnly)
CALIFORNIA INSTITUTE OF TECHNOLOGY [US/US]; 1200 E. California Blvd, MS 201-85 Pasadena, CA 91125, US (AllExceptUS)
Inventors:
DAVIS, Mark, E.; US
Agent:
VINCENT, Matthew, P. ; Fish and Neave IP Group of Ropes & Gray LLP One International Place Boston, MA 02110-2624, US
Priority Data:
11/044,67727.01.2005US
Title (EN) INHIBITOR NUCLEIC ACIDS
(FR) ACIDES NUCLEIQUES INHIBITEURS
Abstract:
(EN) The present invention provides methods and compositions for attenuating expression of a target gene in vivo. In general, the method includes administering RNAi constructs (such as small-interfering RNAs (i.e., siRNAs) that are targeted to particular mRNA sequences, or nucleic acid material that can produce siRNAs in a cell), in an amount sufficient to attenuate expression of a target gene by an RNA interference mechanism. In particular, the RNAi constructs may include one or more modifications to improve serum stability, cellular uptake and/or to avoid non- specific effect. In certain embodiments, the RNAi constructs contain an aptamer portion. The aptamer may bind to human serum albumin to improve serum half life. The aptamer may also bind to a cell surface protein that improves uptake of the construct.
(FR) La présente invention concerne des méthodes et des compositions permettant d'atténuer l'expression d'un gène cible in vivo. En général, la méthode consiste à administrer des constructions d'ARNi (telles que des petits ARN interférants (à savoir, des ARNsi) qui sont ciblés pour spécifier des séquences d'ARNm ou de la matière d'acide nucléique qui peut produire des ARNsi dans une cellule), dans une quantité suffisante afin d'atténuer l'expression d'un gène cible par le biais d'un mécanisme d'interférence d'ARN. Notamment, les constructions d'ARNi peuvent comprendre au moins une modification de façon à améliorer la stabilité du sérum, l'assimilation cellulaire ou à éviter un effet non spécifique. Dans certains modes de réalisation, les constructions d'ARNi renferment une partie d'aptamère. L'aptamère peut se lier à l'albumine du sérum humain afin d'améliorer la demi-vie du sérum. L'aptamère peut, aussi, se lier à une protéine de surface cellulaire qui améliore l'assimilation de la construction.
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Designated States: AE, AG, AL, AM, AT, AU, AZ, BA, BB, BG, BR, BW, BY, BZ, CA, CH, CN, CO, CR, CU, CZ, DE, DK, DM, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, HR, HU, ID, IL, IN, IS, JP, KE, KG, KM, KN, KP, KR, KZ, LC, LK, LR, LS, LT, LU, LV, LY, MA, MD, MG, MK, MN, MW, MX, MZ, NA, NG, NI, NO, NZ, OM, PG, PH, PL, PT, RO, RU, SC, SD, SE, SG, SK, SL, SM, SY, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, YU, 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 Office (AM, AZ, BY, KG, KZ, MD, RU, TJ, TM)
European Patent Office (EPO) (AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HU, IE, IS, IT, LT, LU, LV, MC, NL, 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)