AGENT FOR TREATMENT OF HUMAN BODY STATES RELATED TO P4HA1 GENE REDUCED EXPRESSION AND/OR REDUCED QUANTITY OF PROLYL 4-HYDROXYLASE ALPHA 1 PROTEIN ON BASIS OF GENE-THERAPEUTIC SUBSTANCES WITH P4HA1 GENE, METHOD OF MANUFACTURE AND OPERATION
Obshchestvo s ogranichennoj otvetstvennostyu "Medservis"
发明人:
Krok Igor Semenovich (RU),Крок Игорь Семенович (RU),Savelieva Natalia (AT),Савелиева Наталиа (AT)
申请号：
RU2017134475
公开号：
RU0002658428C9
申请日:
2017.10.03
申请国别(地区):
RU
年份:
2018
代理人:
摘要：
FIELD: biology.SUBSTANCE: invention relates to molecular biology, biotechnology, genetic engineering and medicine and is a means for treating conditions of the human body associated with a decrease in the expression of the P4HA1 gene and/or a decrease in the amount of protein spilled 4-hydroxylase alpha 1, based on gene therapy substances with the P4HA1 gene, at least one gene therapeutic substance selected from the group of gene therapeutic substances, each representing a genetic construct based on a vector plasmid comprising the cDNA of the P4HA1 gene, with the coding sequence of the protein spilled 4-hydroxylase alpha 1, with deletions 5'- and 3'-non-translated regions, namely the unmodified cDNA gene obtained from the site P4HA1 SEQ ID No: 1, or a modified cDNA of the P4HA1 gene, wherein the modified cDNA of the P4HA1 gene is SEQ ID No: 2, or SEQ ID No: 3, or SEQ ID No: 4, or SEQ ID No: 5, or SEQ ID No: 6, or SEQ ID No: 7, or a combination of these genetic constructs, each of which also contains regulatory elements that provide a high level of expression of the P4HA1 gene in eukaryotic cells.EFFECT: invention effectively increases the amount of protein spilled 4-hydroxylase alpha 1 in eukaryotic cells and to obtain a highly effective agent for the treatment of human body conditions associated with a decrease in the level of expression of the P4HA1 gene and/or a decrease in the amount of the prolyl 4-hydroxylase alpha 1 protein.7 cl, 20 dwg, 18 exИзобретение относится к молекулярной биологии, биотехнологии, генной инженерии и медицине и представляет собой средство для лечения состояний человеческого организма, связанных с уменьшением экспрессии гена Р4НА1 и/или уменьшением количества белка пролил 4-гидроксилазы альфа 1, на основе генно-терапевтических субстанций с геном Р4НА1, представляющее собой, по крайней мере, одну генно-терапевтическую субстанцию, выбранную из группы генно-терапевтических субстанций, каждая из которых представляет генетическую конструкцию н
