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1. WO2020139152 - GENE THERAPY DNA VECTOR AND ITS APPLICATION

Publication Number WO/2020/139152
Publication Date 02.07.2020
International Application No. PCT/RU2019/000990
International Filing Date 20.12.2019
IPC
C12N 15/12 2006.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
12Genes encoding animal proteins
C12N 15/70 2006.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
70Vectors or expression systems specially adapted for E. coli
C12N 1/21 2006.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
1Microorganisms, e.g. protozoa; Compositions thereof; Processes of propagating, maintaining or preserving microorganisms or compositions thereof; Processes of preparing or isolating a composition containing a microorganism; Culture media therefor
20Bacteria; Culture media therefor
21modified by introduction of foreign genetic material
A61K 48/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
C12R 1/19 2006.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
RINDEXING SCHEME ASSOCIATED WITH SUBCLASSES C12C-C12Q75
1Microorganisms
01Bacteria or actinomycetales
185Escherichia
19Escherichia coli
CPC
A61K 48/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
A61K 48/005
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
005characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
C12N 15/70
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
70Vectors or expression systems specially adapted for E. coli
C12R 1/19
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
RPROCESSES USING MICROORGANISMS
1Processes using microorganisms
01using bacteria or actinomycetales
185Escherichia
19Escherichia coli
Applicants
  • CELL END GENE THERAPY LTD [GB]/[GB]
  • OBSCHESTVO S OGRANICHENNOI OTVETSTVENNOSTJU "PRORYVNYE INNOVATSIONNYE TEKHNOLOGII" [RU]/[RU]
Inventors
  • SAVELIEVA, Natalia
Agents
  • KOVALENKO, Valentina Vasilievna
Priority Data
201814674226.12.2018RU
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) GENE THERAPY DNA VECTOR AND ITS APPLICATION
(FR) VECTEUR D'ADN DE THÉRAPIE GÉNIQUE ET SON APPLICATION
Abstract
(EN)
The invention refers to genetic engineering and can be used in biotechnology, medicine, and agriculture for the manufacture of gene therapy products. Gene therapy DNA vector based on the gene therapy DNA vector VTvaflV carrying the therapeutic gene selected from the group of KRT5, KRT14, LAMB 3, and COL7A1 genes was constructed in order to increase the expression level of this therapeutic gene in humans and animals, while gene therapy DNA vector VTvafl7-KRT5, or VTvafl7-KRTl4, or VTvafl7-LAMB3, or VTvafl7-COL7Al has the nucleotide sequence SEQ ID No. 1, or SEQ ID No. 2, or SEQ ID No. 3, or SEQ ID No. 4, respectively. The gene therapy DNA vector contains no nucleotide sequences of viral origin and no antibiotic resistance genes, which ensures its safe use for gene therapy in humans and animals. A method of obtaining the specified vector, the use of the vector, a strain of Escherichia coli carrying the specified vector, and a method of industrial production of the specified vector are also provided.
(FR)
La présente invention se rapporte à l'ingénierie génétique et peut être utilisée en biotechnologie, en médecine et en agriculture pour la fabrication de produits de thérapie génique. Un vecteur d'ADN de thérapie génique basé sur le vecteur d'ADN de thérapie génique VTvaflV portant le gène thérapeutique choisi parmi le groupe des gènes KRT5, KRT14, LAMB3, et COL7A1 a été construit afin d'augmenter le niveau d'expression de ce gène thérapeutique chez l'homme et l'animal, tandis que le vecteur d'ADN de thérapie génique VTvafl7-KRT5, ou VTvafl7-KRTl4, ou VTvafl7-LAMB3, ou VTvafl7-COL7Al présente la séquence nucléotidique SEQ ID No. 1, ou SEQ ID No. 2, Ou SEQ ID No. 3, Ou SEQ ID No. 4, respectivement. Le vecteur d'ADN de thérapie génique ne contient pas de séquences nucléotidiques d'origine virale ni de gènes de résistance aux antibiotiques, assurant son utilisation sûre pour une thérapie génétique chez l'homme et l'animal. L'invention concerne également un procédé d'obtention du vecteur spécifié, l'utilisation du vecteur, une souche d'Escherichia coli portant le vecteur spécifié, et un procédé de production industrielle du vecteur spécifié.
Also published as
Latest bibliographic data on file with the International Bureau