Processing

Please wait...

PATENTSCOPE will be unavailable a few hours for maintenance reason on Tuesday 27.07.2021 at 12:00 PM CEST
Settings

Settings

Goto Application

1. WO2020139156 - GENE THERAPY DNA VECTOR AND ITS APPLICATION

Publication Number WO/2020/139156
Publication Date 02.07.2020
International Application No. PCT/RU2019/000996
International Filing Date 23.12.2019
IPC
C12N 15/12 2006.1
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 2006.1
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 2006.1
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 2006.1
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 2006.1
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 48/005
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 15/102
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
10Processes for the isolation, preparation or purification of DNA or RNA
102Mutagenizing nucleic acids
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
C12N 15/85
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
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
C12N 15/88
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
87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
88using microencapsulation, e.g. using ; amphiphile; liposome vesicle
C12N 2310/20
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
2310Structure or type of the nucleic acid
10Type of nucleic acid
20involving clustered regularly interspaced short palindromic repeats [CRISPRs]
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
201814708327.12.2018RU
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 is related to genetic engineering and can be used in biotechnology, medical science and agriculture to create gene therapy medicinal products. Gene therapy DNA vector based on the VTvaflV gene therapy DNA vector is proposed, the vector carrying the target Cas9 gene for heterologous expression of this target gene in human and animal cells upon implementation of various genome editing methods. Gene therapy DNA vector VTvafl7-Cas9 having a nucleotide sequence SEQ ID No. 1. Gene therapy DNA vector based on gene therapy DNA vector VTvaflV carrying the Cas9 target gene, said DNA vector is unique due to the fact that the constructed gene therapy DNA vector VTvafl7-Cas9, due to the limited size of VTvafl7 vector part not exceeding 3200 bp, has the ability to efficiently penetrate into human and animal cells and express the Cas9 target gene cloned to it. A method of production of the specified vector, the method of usage of the vector, Escherichia coli strain carrying the specified vector, as well as a method of production of the specified vector on an industrial scale are also provided.
(FR)
L'invention se rapporte au génie génétique et peut être utilisée dans la biotechnologie, la science médicale et l'agriculture pour créer des produits médicinaux de thérapie génique. L'invention concerne un vecteur d'ADN de thérapie génique basé sur le vecteur d'ADN de thérapie génique VTvaflV, le vecteur portant le gène Cas9 cible pour l'expression hétérologue dudit gène cible dans des cellules humaines et animales lors de la mise en oeuvre de divers procédés d'édition de génome. Le vecteur d'ADN de thérapie génique VTvafl7-Cas9 possède une séquence nucléotidique SEQ ID No.1. Le vecteur d'ADN de thérapie génique basé sur un vecteur d'ADN de thérapie génique VTvaflV portant le gène cible Cas9 est unique en raison du fait que le vecteur d'ADN de thérapie génique construit VTvafl7-Cas9, grâce à la taille limitée de la partie de vecteur VTvafl7 ne dépassant pas 3200 bp, possède la capacité de pénétrer efficacement dans les cellules humaines et animales et d'exprimer le gène cible Cas9 lui étant cloné. L'invention concerne également un procédé de production du vecteur spécifié, le procédé d'utilisation dudit vecteur, une souche d'Escherichia coli portant le vecteur spécifié, ainsi qu'un procédé de production du vecteur spécifié à l'échelle industrielle.
Also published as
Latest bibliographic data on file with the International Bureau