Patent RU-2734726-C1 (RU)
Title: Gene-therapeutic dna vector based on the gene-therapeutic dna vector gdtt1_8nas12, carrying the target gene selected from a group of genes ddc, il10, il13, ifnb1, tnfrsf4, tnfsf10, bcl2, hgf, il2 to increase the expression level of said target genes, a method for production and use thereof, a strain escherichia coli jm110-nas/gdtt1_8nas12-ddc or escherichia coli jm110-nas/gdtt1_8nas12-il10 or escherichia coli jm110-nas/gdtt1_8nas12-il13 or escherichia coli jm110-nas/gdtt1_8nas12-ifnb1 or escherichia coli jm110-nas/gdtt1_8nas12-tnfrsf4 or escherichia coli jm110-nas/gdtt1_8nas12-tnfsf10 or escherichia coli jm110-nas/gdtt1_8nas12-bcl2 or escherichia coli jm110-nas/gdtt1_8nas12-hgf or escherichia coli jm110-nas/gdtt1_8nas12-il2, carrying a gene-therapeutic dna vector, method for production thereof, a method for industrial production of a gene-therapeutic dna vector
Inventor: Gamolski Anton
IPC: C12N15/70
Filed: 2020-01-15
Granted: 2020-10-22
Abstract: FIELD: genetic engineering. 
     SUBSTANCE: invention refers to genetic engineering and can be used in biotechnology, medicine and agriculture to develop gene therapy preparations. A gene-therapeutic DNA vector based on the gene-therapeutic DNA vector GDTT1.8NAS12 is created for treating the diseases characterized by the progressive pathological change of the nervous tissue structure and neuron function, including their death, associated with genetic factors, including mutations in genes coding proteins critical for normal functioning of neurons, including Huntington's disease, inherited forms of amyotrophic lateral sclerosis, as well as with disturbed coagulation of tertiary structure of proteins, including Parkinson's disease, Alzheimer's disease, with injuries of central nervous system, with disturbed oxygen supply of brain or spinal cord, with deviations in energy metabolism of neurons and axonal transport or with autoimmune demyelinating processes, including multiple sclerosis, wherein the gene-therapeutic DNA vector comprises a coding portion of the targeted DDC gene cloned into the gene-therapeutic DNA vector GDTT1.8NAS12, to obtain a gene-therapeutic DNA vector GDTT1.8NAS12-DDC, with nucleotide sequence SEQ ID No. 1, or IL10, cloned into a gene-therapeutic DNA vector GDTT1.8NAS12, to obtain a gene-therapeutic DNA vector GDTT1.8NAS12-IL10, with nucleotide sequence SEQ ID No. 2, or IL13, cloned into a gene-therapeutic DNA vector GDTT1.8NAS12, to obtain a gene-therapeutic DNA vector GDTT1.8NAS12-IL13, with nucleotide sequence SEQ ID No. 3, or IFNB1, cloned into a gene-therapeutic DNA vector GDTT1.8NAS12, to obtain a gene-therapeutic DNA vector GDTT1.8NAS12-IFNB1, with nucleotide sequence SEQ ID No. 4, or TNFRSF4, cloned into a gene-therapeutic DNA vector GDTT1.8NAS12, to obtain a gene-therapeutic DNA vector GDTT1.8NAS12-TNFRSF4, with nucleotide sequence SEQ ID No. 5, or TNFSF10, cloned into a gene-therapeutic DNA vector GDTT1.8NAS12, to obtain a gene-therapeutic DNA vector GDTT1.8NAS12-TNFSF10, with nucleotide sequence SEQ ID No. 6, or BCL2, cloned into a gene-therapeutic DNA vector GDTT1.8NAS12, to obtain a gene-therapeutic DNA vector GDTT1.8NAS12-BCL2, with nucleotide sequence SEQ ID No. 7, or HGF, cloned into a gene-therapeutic DNA vector GDTT1.8NAS12, to obtain a gene-therapeutic DNA vector GDTT1.8NAS12-HGF, with nucleotide sequence SEQ ID No. 8, or IL2, cloned into a gene-therapeutic DNA vector GDTT1.8NAS12, to obtain a gene-therapeutic DNA vector GDTT1.8NAS12-IL2, with nucleotide sequence SEQ ID No. 9. 
     EFFECT: gene-therapeutic DNA-vector for treating diseases characterized by progressive pathological change in the structure of nerve tissue and neuron function. 
     24 cl, 25 dwg, 35 ex