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

FIELD: biotechnology.

SUBSTANCE: following is described: 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 ITR sequences are located, within the AAV ITR 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. The following is also described: 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 ITR sequences are located ; within the AAV ITR, the eukaryotic polymerase III promoter — U6 is sequentially located, after which there is a guide RNA expression cassette, including a guide RNA sequence in crRNA format, 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 the control of which The protein-coding sequence of the hCYP21A2 gene is 78–99% identical to the 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: group of inventions expands the arsenal of means for producing proteins in mammalian cells.

9 cl, 3 dwg, 4 tbl, 4 ex