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1. (WO2017096053) METHODS OF REDUCING EXPRESSION OF X-INACTIVATION ESCAPEE GENES AND AUTOSOMAL GENES
Latest bibliographic data on file with the International Bureau

Pub. No.: WO/2017/096053 International Application No.: PCT/US2016/064438
Publication Date: 08.06.2017 International Filing Date: 01.12.2016
IPC:
C12N 15/113 (2010.01)
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
113
Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
Applicants:
THE GENERAL HOSPITAL CORPORATION [US/US]; 55 Fruit Street Boston, Massachusetts 02114, US
Inventors:
LEE, Jeannie T.; US
CHU, Hsueh-Ping; US
Agent:
DEYOUNG, Janice Kugler; US
FASSE, J. Peter; US
Priority Data:
62/261,69801.12.2015US
Title (EN) METHODS OF REDUCING EXPRESSION OF X-INACTIVATION ESCAPEE GENES AND AUTOSOMAL GENES
(FR) PROCÉDÉS DE RÉDUCTION DE L'EXPRESSION DE GÈNES FUGITIFS DE L'INACTIVATION DU CHROMOSOME X ET GÈNES AUTOSOMIQUES
Abstract:
(EN) Inhibitory nucleic acids, e.g., antisense oligonucleotides (ASO) against PAR-TERRA RNA or other chromosome-specific TERRA transcripts (i.e., inclusive of chromosome-specific subtelomeric sequences), and methods of use thereof to downregulate expression of escapee genes on the inactive X chromosome, expression from the active X chromosome, subtelomeric autosomal loci (e.g., FSHD locus), or expression of autosomal genes involved in growth control and apoptosis, e.g., in cells and subjects with supernumerary X chromosomes and/or cancer and other human diseases.
(FR) L'invention concerne des acides nucléiques inhibiteurs, par exemple des oligonucléotides antisens contre l'ARN PAR-TERRA ou d'autres transcrits de TERRA spécifiques des chromosomes (c'est-à-dire, incluant des séquences subtélomériques spécifiques de chromosome), et des procédés d'utilisation associés pour réguler négativement l'expression de gènes fugitifs sur le chromosome X inactif, l'expression du chromosome X actif, des locus autosomiques subtélomériques (par exemple, le locus FSHD), ou l'expression de gènes autosomiques impliqués dans la régulation de la croissance et l'apoptose, par exemple dans des cellules et des sujets portant des chromosomes X surnuméraires et/ou un cancer et d'autres maladies humaines.
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, DJ, 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, KW, KZ, LA, LC, LK, LR, LS, 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, ST, 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)
Also published as:
US20180346906