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1. (WO2018203092) GENE THERAPY FOR CILIOPATHIES
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Pub. No.: WO/2018/203092 International Application No.: PCT/GB2018/051219
Publication Date: 08.11.2018 International Filing Date: 04.05.2018
IPC:
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
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:
UCL BUSINESS PLC [GB/GB]; The Network Building 97 Tottenham Court Road London W1T 4TP, GB
Inventors:
BEALES, Phillip; GB
HERNANDEZ, Victor; GB
Agent:
WITHERS & ROGERS LLP; 4 More London Riverside London SE1 2AU, GB
Priority Data:
1707212.505.05.2017GB
Title (EN) GENE THERAPY FOR CILIOPATHIES
(FR) THÉRAPIE GÉNIQUE CONTRE LES CILIOPATHIES
Abstract:
(EN) There is described a vector for treating a ciliopathy such as Bardet-Biedl syndrome, wherein the vector comprises a promoter operably linked to a ciliopathy gene, wherein the vector can provide transduction of the ciliopathy gene into multiple organs, wherein the promoter is a ubiquitous promoter which can provide expression of the ciliopathy gene in the transduced organs, and wherein the ciliopathy gene encodes a functional protein corresponding to the protein that is mutated in the ciliopathy. Also described is the use of the above vector in a method of treating a ciliopathy, the method comprising administering a therapeutically effective amount of the vector to a patient suffering from a ciliopathy.
(FR) L'invention concerne un vecteur pour le traitement d'une ciliopathie telle que le syndrome de Bardet-Biedl, le vecteur comprenant un promoteur lié de manière fonctionnelle à un gène associé aux ciliopathies, le vecteur pouvant fournir une transduction du gène associé aux ciliopathies dans de multiples organes, le promoteur étant un promoteur ubiquitaire qui peut permettre l'expression du gène associé aux ciliopathies dans les organes transduits, et le gène associé aux ciliopathie codant pour une protéine fonctionnelle correspondant à la protéine qui est mutée dans la ciliopathie. L'invention concerne également l'utilisation du vecteur ci-dessus dans un procédé de traitement d'une ciliopathie, le procédé comprenant l'administration d'une quantité thérapeutiquement efficace du vecteur à un patient souffrant d'une ciliopathie.
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)