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1. (WO2004033620) METHODS AND COMPOSITIONS FOR THERAPEUTIC USE OF RNA INTERFERENCE
Latest bibliographic data on file with the International Bureau

Pub. No.: WO/2004/033620 International Application No.: PCT/US2002/035453
Publication Date: 22.04.2004 International Filing Date: 04.11.2002
Chapter 2 Demand Filed: 28.05.2003
IPC:
A61K 9/00 (2006.01) ,A61K 9/127 (2006.01) ,A61K 9/16 (2006.01) ,A61K 48/00 (2006.01) ,C12N 15/87 (2006.01)
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9
Medicinal preparations characterised by special physical form
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9
Medicinal preparations characterised by special physical form
10
Dispersions; Emulsions
127
Liposomes
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9
Medicinal preparations characterised by special physical form
14
Particulate form, e.g. powders
16
Agglomerates; Granulates; Microbeadlets
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
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
87
Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
Applicants:
INSERT THERAPEUTICS, INC. [US/US]; 2585 Nina Street Pasadena, CA 91107, US (AllExceptUS)
DAVIS, Mark, E. [US/US]; US (UsOnly)
JENSEN, Gregory, S. [US/US]; US (UsOnly)
PUN, Suzie, Hwang [US/US]; US (UsOnly)
Inventors:
DAVIS, Mark, E.; US
JENSEN, Gregory, S.; US
PUN, Suzie, Hwang; US
Agent:
VINCENT, Matthew, P.; Ropes & Gray One International Place Boston, MA 02110-2624, US
Priority Data:
60/33631402.11.2001US
60/337,30405.11.2001US
60/418,90915.10.2002US
Title (EN) METHODS AND COMPOSITIONS FOR THERAPEUTIC USE OF RNA INTERFERENCE
(FR) PROCEDES ET COMPOSITIONS PERMETTANT L'UTILISATION THERAPEUTIQUE DE L'INTERFERENCE ARN
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, e.g., in a sequence-dependent, PKR-independent manner. In particular, the subject method can be used to alter the growth, survival or differentiation of cells for therapeutic and cosmetic purposes.
(FR) L'invention concerne des procédés et des compositions permettant d'atténuer l'expression d'un gène cible in vivo. En général, ce procédé consiste à administrer des RNAi recombinés (tels que des petits ARN interférants (ou siRNA) ciblés sur des séquences particulières d'ARNm, ou des acides nucléiques capables de produire des siARN dans une cellule), à raison d'une quantité suffisante pour atténuer l'expression d'un gène cible par un mécanisme d'interférence ARN, autrement dit d'une manière dépendante de la séquence, et indépendante de PKR. Le procédé décrit permet en particulier de modifier la croissance, la survie ou la différenciation des cellules à des fins thérapeutiques et cosmétiques.
Designated States: AE, AG, AL, AM, AT, AU, AZ, BA, BB, BG, BR, BY, BZ, CA, CH, CN, CO, CR, CU, CZ, DE, DK, DM, DZ, EC, EE, ES, FI, GB, GD, GE, GH, GM, HR, HU, ID, IL, IN, IS, JP, KE, KG, KP, KR, KZ, LC, LK, LR, LS, LT, LU, LV, MA, MD, MG, MK, MN, MW, MX, MZ, NO, NZ, OM, PH, PL, PT, RO, RU, SD, SE, SG, SK, SL, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VN, YU, ZA, ZM, ZW
African Regional Intellectual Property Organization (ARIPO) (GH, GM, KE, LS, MW, MZ, 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, IE, IT, LU, MC, NL, PT, SE, 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)
Also published as:
IL161733EP1575976EP2325193JP2005527639CN1771255CA2465860
AU2002368202