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1. (WO2017201121) RECOMBINANT AAV FOR GENE THERAPY IN LUNGS
Latest bibliographic data on file with the International Bureau    Submit observation

Pub. No.:    WO/2017/201121    International Application No.:    PCT/US2017/033020
Publication Date: 23.11.2017 International Filing Date: 17.05.2017
IPC:
A61K 35/76 (2015.01), A61K 48/00 (2006.01), C12N 7/00 (2006.01), C12N 15/86 (2006.01)
Applicants: UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INC. [US/US]; 223 Grinter Hall Gainesville, FL 32611 (US)
Inventors: ASLANDIDI, George, Vladimirovich; (US).
COLON-CORTES, Yanerys, M.; (US).
ABU-HASAN, Mutasim, N.; (US)
Agent: WALLER, Patrick, R.H.; (US)
Priority Data:
62/337,847 17.05.2016 US
Title (EN) RECOMBINANT AAV FOR GENE THERAPY IN LUNGS
(FR) VAA RECOMBINÉ POUR LA THÉRAPIE GÉNIQUE DES POUMONS
Abstract: front page image
(EN)Gene therapy is an attractive approach to treat genetic lung disorders, particular cystic fibrosis (CF), by treating the cause of the disease by restoring the defective gene. Herein are disclosed compositions of variant adeno-associated virus (AAV) with mutations in capsid protein and methods of delivering a gene to lung tissue of a host using the variant AAV.
(FR)La thérapie génique est une approche intéressante pour traiter des troubles génétiques pulmonaires, en particulier la fibrose kystique (CF), en traitant la cause de la maladie par la restauration du gène défectueux. L'invention concerne des compositions de variants de virus adéno-associés (VAA) présentant des mutations dans la protéine de capside et des méthodes d'administration d'un gène aux tissus pulmonaires d'un hôte à l'aide du variant de VAA.
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 (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)