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1. (WO2019063747) MUTATED ADENO-ASSOCIATED VIRAL CAPSID PROTEINS FOR CHEMICAL COUPLING OF LIGANDS, NANOPARTICLES OR DRUGS VIA THIOETHER BINDING AND PRODUCTION METHOD THEREOF
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Pub. No.: WO/2019/063747 International Application No.: PCT/EP2018/076377
Publication Date: 04.04.2019 International Filing Date: 28.09.2018
IPC:
C07K 14/015 (2006.01) ,C12N 7/00 (2006.01) ,C12N 15/86 (2006.01) ,C12N 15/35 (2006.01) ,C12N 15/63 (2006.01) ,C12N 5/10 (2006.01) ,A61K 35/76 (2015.01) ,A61K 48/00 (2006.01)
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
K
PEPTIDES
14
Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
005
from viruses
01
DNA viruses
015
Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
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
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
31
Genes encoding microbial proteins, e.g. enterotoxins
33
Genes encoding viral proteins
34
Proteins from DNA viruses
35
Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
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
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
5
Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
10
Cells modified by introduction of foreign genetic material, e.g. virus-transformed cells
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
Applicants:
UNIVERSITÄT ZU KÖLN [DE/DE]; Albertus-Magnus-Platz 50923 Köln, DE
Inventors:
BÜNING, Hildegard; DE
HUBER, Anke; DE
PERABO, Luca; DE
Agent:
GRAMM, LINS & PARTNER PATENT- UND RECHTSANWÄLTE PARTGMBB; Freundallee 13a 30173 Hannover, DE
Priority Data:
17193742.828.09.2017EP
Title (EN) MUTATED ADENO-ASSOCIATED VIRAL CAPSID PROTEINS FOR CHEMICAL COUPLING OF LIGANDS, NANOPARTICLES OR DRUGS VIA THIOETHER BINDING AND PRODUCTION METHOD THEREOF
(FR) PROTÉINES CAPSIDIQUES VIRALES ADÉNO-ASSOCIÉES MUTÉES POUR COUPLAGE CHIMIQUE DE LIGANDS, DE NANOPARTICULES OU DE MÉDICAMENTS PAR LIAISON THIOÉTHER ET LEUR PROCÉDÉ DE PRODUCTION
Abstract:
(EN) In a first aspect, the present invention relates to a mutated adeno-associated virus (AAV) capsid protein or fragment thereof wherein a substitution of a wild type non-cysteine amino acid into a cysteine is present whereby the wild type non-cysteine amino acid is exposed on the outer surface of the capsid of an AAV particle. In a further aspect, a mutated AAV particle comprising the AAV capsid protein or fragment thereof according to the present invention is provided. In addition, a nucleic acid encoding the AAV capsid protein according to the present invention is identified together with a corresponding nucleic acid vector, in particular, a plasmid or a gene string. In addition, a host cell containing the nucleic acid vector or the nucleic acid according to the present invention as well as a composition comprising at least an infectious (transducing) AAV particle containing a mutated AAV capsid protein as defined herein together with a non-infectious AAV particle containing a mutated AAV capsid protein as e.g. defined herein is disclosed. Further, a method for the modification of a mutated AAV particle is disclosed allowing a specific modification of the same including an embodiment using a reagent addressing the cysteine residues for binding reaction such as a thioether binding.
(FR) Selon un premier aspect, la présente invention concerne une protéine capsidique d'un virus adéno-associé (AAV) mutée ou un fragment de celle-ci, une substitution d'un acide aminé non cystéine de type sauvage en une cystéine étant présente, de sorte que l'acide aminé non-cystéine de type sauvage est exposé sur la surface externe de la capside d'une particule d'un AAV. Selon un autre aspect, l'invention concerne une particule d'un AAV mutée comprenant la protéine capsidique du AAV ou un fragment de celle-ci selon la présente invention. De plus, un acide nucléique codant pour la protéine capsidique du AAV selon la présente invention est identifié conjointement avec un vecteur d'acide nucléique correspondant, en particulier, un plasmide ou une chaîne de gènes L'invention concerne également, une cellule hôte contenant le vecteur d'acide nucléique ou l'acide nucléique ainsi qu'une composition comprenant au moins une particule infectieuse du AAV (transductrice) incorporant une protéine capsidique du AAV mutée selon l'invention conjointement avec une particule du AAV non infectieuse incorporant une protéine capsidique du AAV mutée, par exemple, telle que définie dans la présente invention. L'invention concerne en outre un procédé de modification d'une particule d'un AAV mutée permettant une modification spécifique de celle-ci comprenant un mode de réalisation utilisant un réactif adressant les résidus de cystéine pour une réaction de liaison telle qu'une liaison thioéther.
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)