FIELD: biotechnologies.
SUBSTANCE: invention relates to the biotechnology. Described is an adeno-associated virus (AAV) factor VIII (FVIII) expression vector comprising a nucleic acid containing 5'-inverted end repeat (ITR) of AAV2, a liver-specific transcription regulation region, codon-optimized region coding functionally active FVIII, possibly one or more introns, a polyadenylation sequence and 3'-ITR AAV2, wherein the region coding functionally active FVIII includes nucleotides 923–5296 of SEQ ID NO: 9 and wherein vector has length less than 5000 nucleotides. Also described is a method of producing a particle of recombinant adeno-associated virus (AAV), involving: A) culturing the cell that has been transfected with the AAV expression vector FVIII described above; and B) obtaining a recombinant AAV particle from a supernatant of a cultured cell, wherein the cultured cell further comprises one or more vectors expressing helper functions required for packing the AAV particles. Also described is a particle of recombinant adeno-associated virus (AAV) for treating haemophilia A, prepared by said method, wherein the AAV particle comprises a genome comprising a nucleic acid containing 5'-inverted terminal repeat (ITR) of AAV, a liver-specific transcription regulation region, a codon-optimized region encoding a functionally active FVIII, optionally one or more introns, a polyadenylation sequence and 3'-ITR AAV2, wherein the region coding functionally active FVIII includes nucleotides 923–5296 of the sequence SEQ ID NO: 9 and wherein vector has length less than 5000 nucleotides. Also presented is a method of treating a patient suffering from haemophilia.
EFFECT: invention extends the range of products for treating haemophilia A.
16 cl, 10 dwg, 1 tbl, 6 ex
