Gene therapy DNA vector based on gene therapy DNA vector GDTT1.8NAS12 carrying HFE therapeutic gene for enhanced expression of the therapeutic gene, method of its production and use, Escherichia coli strain JM110-NAS/GDTT1.8NAS12-HFE carrying the gene therapy DNA vector, method of its production, method of the gene therapy DNA vector production on an industrial scale

Abstract: proposed is a gene-therapy dna vector based on gene-therapy dna vector gdtt1.8nas12 for treating diseases characterized by impaired functioning of the hfe protein responsible for regulating iron metabolism in the human body, and for treating diseases related to impaired expression of the hfe gene, inter alia diseases caused by insufficient expression of the hfe gene and/or by the presence of mutations in the hfe gene, inter alia in the case of haemochromatosis, wherein the gene-therapy dna vector contains the coding part of the target gene hfe, cloned in gene-therapy dna vector gdtt1.8nas12 to produce gene-therapy dna vector gdtt1.8nas12-hfe having the nucleotide sequence seq id no: 1. the resulting gene-therapy dna vector gdtt1.8nas12-hfe is capable of effectively penetrating into human and animal cells and expressing the target gene cloned in it, i.e. hfe, by virtue of the limited size of the vector part gdtt1.8nas12, which is not greater than 2600 bp. in the proposed gene-therapy dna vector gdtt1.8nas12-hfe, nucleotide sequences which are not antibiotic resistance genes, viral genes or regulatory elements of viral genomes are used as structural elements, thus allowing the gene-therapy dna vector to be used safely for gene therapy in humans and animals.

Inventor(s): Anton GAMOLSKI

CPC Classification: A61K48/005 (Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy)

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