FIELD: biotechnology; medicine.
SUBSTANCE: invention relates to biotechnology and medicine and represents a gene-therapeutic DNA vector based on the gene-therapeutic DNA vector GDTT1.8NAS12 for treating diseases characterized by disturbed functions of HFE protein responsible for regulation of iron metabolism in the human body, for therapy of diseases associated with disturbed expression of the HFE gene, including those caused by insufficient expression of the HFE gene and/or presence of mutations in the HFE gene, including in hemachromatosis, wherein the gene-therapeutic DNA vector contains a coding portion of the target HFE gene cloned into the gene-therapeutic DNA vector GDTT1.8NAS12, to obtain a genotyping DNA vector GDTT1.8NAS12-HFE, with the nucleotide sequence SEQ ID No. 1.
EFFECT: created gene-therapeutic DNA vector GDTT1_8NAS12-HFE has the ability to efficiently penetrate into human and animal cells and to express the target HFE gene cloned into it.
8 cl, 7 dwg, 9 ex
Authors
Dates
2020-10-01—Published
2019-12-30—Filed