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1. (WO2011097388) SELECTIVE INHIBITION OF POLYGLUTAMINE PROTEIN EXPRESSION
Latest bibliographic data on file with the International Bureau

Pub. No.: WO/2011/097388 International Application No.: PCT/US2011/023615
Publication Date: 11.08.2011 International Filing Date: 03.02.2011
IPC:
A61K 38/17 (2006.01) ,A61K 31/713 (2006.01) ,C12N 15/11 (2006.01)
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38
Medicinal preparations containing peptides
16
Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
17
from animals; from humans
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
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
Applicants:
ALNYLAM PHARMACEUTICALS, INC. [US/US]; 300 Third Street Cambridge, MA 02142, US (AllExceptUS)
THE BOARD OF REGENTS OF THE UNIVERSITY OF TEXAS SYSTEM [US/US]; 201 West 7th St. Austin, TX 78701, US (AllExceptUS)
COREY, David, R. [US/US]; US (UsOnly)
HU, Jiaxin [CN/US]; US (UsOnly)
SAH, Dinah, W., Y. [US/US]; US (UsOnly)
Inventors:
COREY, David, R.; US
HU, Jiaxin; US
SAH, Dinah, W., Y.; US
Agent:
HIGHLANDER, Steven, L.; Fulbright & Jaworski L.L.P. 600 Congress Ave., Suite 2400 Austin, TX 78701, US
Priority Data:
61/301,06703.02.2010US
61/321,41606.04.2010US
61/417,04824.11.2010US
Title (EN) SELECTIVE INHIBITION OF POLYGLUTAMINE PROTEIN EXPRESSION
(FR) INHIBITION SÉLECTIVE DE L'EXPRESSION DE LA PROTÉINE DE POLYGLUTAMINE
Abstract:
(EN) The present invention relates to the selective inhibition of protein expression of CAG repeat-related disease proteins such as Huntingtin Disease Protein and Ataxin-3 using double- stranded RNAs and nucleic acid analogs. Chemically-modified RNAs having at least one mismatch as compared to the target CAG repeat sequence are specifically contemplated.
(FR) La présente invention a pour objet l'inhibition sélective de l'expression protéique de protéines de maladies associées à la répétition de CAG telles que la protéine de la maladie de Huntington et l'ataxine 3 en utilisant des ARN double brin et des analogues d'acide nucléique. L'invention concerne spécifiquement des ARN modifiés par voie chimique présentant au moins un mésappariement comparativement à la séquence cible de répétitions de CAG.
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, LR, 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 (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)