ADENO-ASSOCIATED VIRAL VECTOR CONSISTING OF CAPSID PROTEINS PHP_B, NUCLEIC ACID CODING SMN PROTEIN, AND USE THEREOF Russian patent published in 2025 - IPC C12N15/09 C12N15/63 C12N15/86 A61K48/00 A61P25/00 

FIELD: biotechnology.

SUBSTANCE: described is an adeno-associated viral (rAAV) vector based on the AAV.PHP.B serotype for delivering the SMN1 gene to the central nervous system, containing a polynucleotide, coding motor neuron survival protein (SMN), characterized by that it has single-stranded gene (ssAAV), capsid includes protein VP1 AAV.PHP.B, having amino acid sequence SEQ ID NO: 1 or amino acid sequence with one or several point mutations, and the expression cassette includes the following elements in direction from 5'-end to 3'-end: ITR; CMV enhancer; CMV promoter SMN1; polyadenylation signal hGH1; ITR. Also described is a recombinant adeno-associated viral (rAAV) vector based on the serotype AAV.PHP.B for delivering the SMN1 gene to the central nervous system, containing a polynucleotide coding a motor neuron survival protein (SMN), characterized by that it has a self-complementary gene (scAAV), capsid includes protein VP1 AAV.PHP.B, having amino acid sequence SEQ ID NO: 1 or amino acid sequence with one or more point mutations, and expression cassette includes the following elements in direction from 5'-end to 3'-end: ITR; CMV enhancer; CB promoter; SV40 intron SMN1; polyadenylation signal BGH; ITR. Disclosed is a pharmaceutical composition for delivering the SMN1 gene to the central nervous system, comprising a recombinant viral vector based on AAV.PHP.B in a combination with one or more pharmaceutically acceptable excipients. Also disclosed is the use of a recombinant viral vector based on AAV.PHP.B or a composition for delivering the SMN1 gene to the central nervous system.

EFFECT: invention extends the range of agents for delivering the SMN1 gene to the central nervous system.

6 cl, 5 dwg, 3 tbl