FIELD: biotechnology.
SUBSTANCE: invention relates to the recombinant production of blood coagulation factor VIII protein with B domain deleted (FVIII-BDD), and can be used in gene therapy for hemophilia A to deliver the FVIII-BDD gene to target cells. Codon-optimized variants of FVIII-BDD-encoding nucleic acids and expression constructs for their delivery to target cells have been proposed.
EFFECT: invention provides a medicinal product with high potential for gene therapy for hemophilia A.
9 cl, 6 dwg, 4 ex
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