SET OF EXPRESSION VECTORS BASED ON ADENO-ASSOCIATED VIRUS Russian patent published in 2024 - IPC C12N15/86 

FIELD: biotechnology.

SUBSTANCE: invention relates to a set of expression vectors for producing proteins in mammalian cells. The set contains an expression vector, which is a viral particle of an adeno-associated virus (AAV) having AAV capsid proteins , in which a single-stranded DNA molecule is packaged as a genome, at the ends of which the AAV ITRs are located, within the AAV ITRs the promoter of the first eukaryotic elongation factor EF1 alpha is sequentially located, histidine tag sequence, intracellular nuclear localization signal 5'-SV40 NLS, codon-optimized sequence of the Streptococcus pyogenes Cas9 gene, another intracellular nuclear localization signal 3'-SV40 NLS, a synthetic polyA signal was used as a transcription terminator, and an expression vector, which is a viral particle of an adeno-associated virus (AAV), having AAV capsid proteins , in which a single-stranded DNA molecule is packaged as a genome, at the ends of which the AAV ITRs are located, inside the eukaryotic promoter of polymerase III — U6, after which the guide RNA expression cassette is located, including a guide RNA sequence in crRNA format, representing a 20-nucleotide sequence complementary to the targeted locus, linked to the trackRNA sequence, a region of homology with the human genome locus AAVS1 is the left homologous arm, a transgene expression cassette: the EFS-NS promoter, under which the protein is located the coding sequence of hCYP21A2 gene is 78–99% identical to the following sequence SEQ ID NO: 3, transcription terminator is polyA signal, region of homology with the human genome locus AAVS1 is the right homologous arm.

EFFECT: invention expands the range of means for the expression of 21-hydroxylase in mammalian cells.

8 cl, 3 dwg, 4 tbl, 4 ex