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1. (WO2019008157) NOVEL POLYNUCLEOTIDES ENCODING A HUMAN FKRP PROTEIN
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Pub. No.: WO/2019/008157 International Application No.: PCT/EP2018/068420
Publication Date: 10.01.2019 International Filing Date: 06.07.2018
IPC:
A61K 38/00 (2006.01) ,A61K 48/00 (2006.01) ,C07K 14/47 (2006.01)
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38
Medicinal preparations containing peptides
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
07
ORGANIC CHEMISTRY
K
PEPTIDES
14
Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435
from animals; from humans
46
from vertebrates
47
from mammals
Applicants:
GENETHON [FR/FR]; 1 bis Rue de l'Internationale 91000 EVRY, FR
UNIVERSITE D'EVRY-VAL-D'ESSONNE [FR/FR]; Boulevard François Mitterrand 91025 EVRY, FR
INSERM (INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE) [FR/FR]; 101 Rue de Tolbiac 75013 PARIS, FR
Inventors:
RICHARD, Isabelle; FR
GICQUEL-ZOUIDA, Evelyne; FR
Agent:
BUCHET, Anne; FR
DENJEAN, Eric; FR
PALIX, Stéphane; FR
VUILLERMOZ, Bruno; FR
SEMAOUNE, Idriss; FR
Priority Data:
17305894.207.07.2017EP
Title (EN) NOVEL POLYNUCLEOTIDES ENCODING A HUMAN FKRP PROTEIN
(FR) NOUVEAUX POLYNUCLÉOTIDES CODANT POUR UNE PROTÉINE FKRP HUMAINE
Abstract:
(EN) The present invention concerns synthetic polynucleotides encoding a human fukutin- related protein (FKRP) wherein said polynucleotides contain at least a mutation avoiding supplementary transcript(s) generated from frameshift start codon(s). Said polynucleotides are useful, especially for treating a pathology linked to a FKRP deficiency or induced by a defect in α-dystroglycan (α-DG) glycosylation, such as LGMD2I.
(FR) La présente invention concerne des polynucléotides synthétiques codant pour une protéine liée à la fukutine (FKRP) humaine, lesdits polynucléotides contenant au moins une mutation évitant un transcrit(s) supplémentaire(s) généré(s) à partir du codon(s) d'initiation de frameshift. Lesdits polynucléotides sont utiles, en particulier pour le traitement d'une pathologie liée à une déficience en FKRP ou induite par un défaut dans la glycosylation de l’α-dystroglycane (α-DG), telle que LGMD2I.
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)