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1. (WO2017153753) ALLELE-SPECIFIC GENE SUPPRESSION
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Pub. No.: WO/2017/153753 International Application No.: PCT/GB2017/050624
Publication Date: 14.09.2017 International Filing Date: 08.03.2017
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:
UCL BUSINESS PLC [GB/GB]; The Network Building 97 Tottenham Court Road London W1T 4TP, GB
Inventors:
MUNTONI, Francesco; GB
ZHOU, Haiyan; GB
REILLY, Mary; GB
Agent:
BENTHAM, Andrew; GB
Priority Data:
1604261.611.03.2016GB
Title (EN) ALLELE-SPECIFIC GENE SUPPRESSION
(FR) SUPPRESSION DE GÈNE SPÉCIFIQUE À UN ALLÈLE
Abstract:
(EN) The invention relates to an oligonucleotide that supresses the expression of an allele carrying a dominant mutation that causes hereditary sensory neuropathy type I (HSNl), wherein the suppression takes place through hybridisation of said oligonucleotide to the DNA of said allele or to an RNA transcript of said allele, and which either does not suppress the expression of a wild-type allele not containing the dominant mutation or suppresses the expression of said wild-type allele to a lesser extent than it suppresses the expression of the dominant mutant allele; and also pharmaceutical compositions comprising oligonucleotides of the invention and treatments of HSNl using such oligonucleotides.
(FR) La présente invention décrit un oligonucléotide qui supprime l’expression d’un allèle portant une mutation dominante qui provoque la neuropathie sensorielle héréditaire de type I (HSN1), la suppression ayant lieu par hybridation dudit oligonucléotide à l’ADN dudit allèle ou à un produit de transcription ARN dudit allèle, et qui soit ne supprime pas l’expression d’un allèle de type sauvage ne contenant pas la mutation dominante, soit supprime l’expression dudit allèle de type sauvage dans une moindre mesure par rapport à la suppression de l’expression d’un allèle mutant dominant ; et également des compositions pharmaceutiques comprenant les oligonucléotides de l’invention et des traitements de la HSN1 en utilisant de tels oligonucléotides.
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, KH, 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 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, KM, ML, MR, NE, SN, TD, TG)
Publication Language: English (EN)
Filing Language: English (EN)