FIELD: biotechnology.
SUBSTANCE: present application relates to biotechnology and can be used in medicine. More specifically, the present invention relates to an isolated codon-optimized nucleic acid which codes a FIX (blood coagulation factor IX) protein, an expression cassette and a vector based thereon, as well as to a recombinant virus based on AAV5 (serotype 5 adeno-associated virus) for increasing FIX gene expression in target cells and their use.
EFFECT: disclosed is a codon-optimized nucleic acid which codes a coagulation factor protein ix, and its use.
10 cl, 5 ex, 1 tbl, 4 dwg
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| CODON-OPTIMIZED NUCLEIC ACID THAT ENCODES B-DOMAIN-DELETED FACTOR VIII PROTEIN AND ITS USE | 2022 |
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RU2793735C2 |
