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1. (WO2017201121) RECOMBINANT AAV FOR GENE THERAPY IN LUNGS
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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)
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
35
Medicinal preparations containing material or reaction products thereof with undetermined constitution
66
Materials from micro-organisms
76
Viruses
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
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
7
Viruses, e.g. bacteriophages; Compositions thereof; Preparation or purification thereof
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
63
Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79
Vectors or expression systems specially adapted for eukaryotic hosts
85
for animal cells
86
Viral vectors
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:
62/337,84717.05.2016US
Title (EN) RECOMBINANT AAV FOR GENE THERAPY IN LUNGS
(FR) VAA RECOMBINÉ POUR LA THÉRAPIE GÉNIQUE DES POUMONS
Abstract:
(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.
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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)