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1. (WO2018100010) ALLELE-SPECIFIC SILENCING THERAPY FOR DYNAMIN 2-RELATED DISEASES
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Pub. No.: WO/2018/100010 International Application No.: PCT/EP2017/080884
Publication Date: 07.06.2018 International Filing Date: 29.11.2017
IPC:
C12N 15/113 (2010.01) ,A61K 31/713 (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
113
Non-coding nucleic acids modulating the expression of genes, e.g. antisense 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
713
Double-stranded nucleic acids or oligonucleotides
Applicants:
ASSOCIATION INSTITUT DE MYOLOGIE [FR/FR]; 47, boulevard de l'Hôpital 75013 PARIS, FR
INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE [FR/FR]; 101 rue de Tolbiac 75013 PARIS, FR
UNIVERSITE PIERRE ET MARIE CURIE (PARIS 6) [FR/FR]; 4, Place Jussieu 75005 PARIS, FR
CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE [FR/FR]; 3 rue Michel Ange 75016 PARIS, FR
Inventors:
BITOUN, Marc; FR
TROCHET, Delphine; FR
PRUDHON, Bernard; FR
Agent:
CABINET BECKER ET ASSOCIES; 25, rue Louis le Grand 75002 PARIS, FR
Priority Data:
16306575.829.11.2016EP
Title (EN) ALLELE-SPECIFIC SILENCING THERAPY FOR DYNAMIN 2-RELATED DISEASES
(FR) TRAITEMENT PAR SILENÇAGE SPÉCIFIQUE D'UN ALLÈLE, DESTINÉ À DES MALADIES ASSOCIÉES À LA DYNAMINE 2
Abstract:
(EN) The invention relates to an allele specific siRNA able to silence the expression of only one allele of a heterozygous DNM2 gene, for treating diseases caused by heterozygous mutation and/or overexpression of Dynamin 2.
(FR) L'invention concerne un ARNsi spécifique d'un allèle, capable de silencer l'expression d'un allèle unique d'un gène DNM2 hétérozygote, dans le but de traiter des maladies provoquées par une mutation hétérozygote et/ou une surexpression de la Dynamine 2.
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, JO, 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)