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1. (WO2019023770) COMPOSITION FOR GENE THERAPY OF THE CENTRAL NERVOUS SYSTEM, PROCESS OF PRODUCTION AND USE THEREOF
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Pub. No.: WO/2019/023770 International Application No.: PCT/BR2018/050236
Publication Date: 07.02.2019 International Filing Date: 12.07.2018
IPC:
A61K 48/00 (2006.01) ,C12N 15/11 (2006.01) ,C12N 15/66 (2006.01) ,A61P 25/00 (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
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
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
66
General methods for inserting a gene into a vector to form a recombinant vector using cleavage and ligation; Use of non-functional linkers or adaptors, e.g. linkers containing the sequence for a restriction endonuclease
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
25
Drugs for disorders of the nervous system
Applicants:
UNIVERSIDADE FEDERAL DO RIO GRANDE DO SUL [BR/BR]; PAULO GAMA, 111 RS 90040060 PORTO ALEGRE, BR
Inventors:
SILVESTRI SCHUH, Roselena; BR
FERREIRA TEIXEIRA, Helder; BR
BALDO, Guilherme; BR
DA SILVEIRA MATTE, Ursula; BR
GIUGLIANI, Roberto; BR
BIDONE, Juliana; BR
Priority Data:
BR 10 2017 016440 331.07.2017BR
Title (EN) COMPOSITION FOR GENE THERAPY OF THE CENTRAL NERVOUS SYSTEM, PROCESS OF PRODUCTION AND USE THEREOF
(FR) COMPOSITION POUR LA THÉRAPIE GÉNIQUE DU SYSTÈME NERVEUX CENTRAL, PROCÉDÉ D’OBTENTION ET UTILISATION DE CELLE-CI
(PT) COMPOSIÇÃO PARA TERAPIA GÊNICA DO SISTEMA NERVOSO CENTRAL, PROCESSO DE OBTENÇÃO E USO DA MESMA
Abstract:
(EN) The present invention describes a composition for gene therapy of the central nervous system comprising nonviral nanoscale (<1.0 micrometer) carriers complexed with at least one nucleic acid for purposes of gene therapy via nasal administration having as the main target the central nervous system, and also the processes for producing said carriers. The present invention pertains to the field of nanotechnology and consists of aqueous formulations that can be used in the pharmaceutical and medical sectors.
(FR) La présente invention concerne une composition pour la thérapie génique du système nerveux central comprenant des supports non viraux de taille nanométrique (< 1,0 micromètre) complexés avec au moins un acide nucléique à des fins de thérapie génique par administration nasale, la cible principale étant le système nerveux central, ainsi que les procédés d'obtention de ces supports. La présente invention relève du domaine de la nanotechnologie et consiste en des formulations aqueuses pouvant être utilisées dans les domaines pharmaceutique et médical.
(PT) A presente invenção descreve uma composição para terapia gênica do sistema nervoso central compreendendo carreadores não-virais de tamanho nanométrico (< 1,0 micrômetro) complexados com ao menos um ácido nucleico para fins de terapia gênica via administração nasal tendo como alvo principal o sistema nervoso central, e ainda os processos de obtenção de tais carreadores. A presente invenção pertence ao campo da nanotecnologia e consiste em formulações aquosas que podem ser utilizadas nas áreas farmacêutica e médica.
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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, 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: Portuguese (PT)
Filing Language: Portuguese (PT)