FIELD: biotechnology.
SUBSTANCE: described is a group of inventions comprising a nucleic acid molecule for expressing a therapeutic protein in a subject, a pharmaceutical composition for treating a disease, deficiency, disorder or condition associated with a coagulation factor, and a method for providing expression of the coagulation factor in a patient. In one of the versions, the nucleic acid molecule contains in direction from 5' to 3': a first ITR, a gene cassette containing a nucleotide coding a therapeutic protein, and a second ITR.
EFFECT: invention extends the range of agents for the expression of the blood coagulation factor in the patient.
18 cl, 4 dwg, 9 tbl, 7 ex
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Authors
Dates
2024-05-14—Published
2018-08-09—Filed