ISOLATED MODIFIED VP1 CAPSID PROTEIN OF ADENO-ASSOCIATED VIRUS OF SEROTYPE 5 (AAV5), CAPSID AND VECTOR BASED ON IT Russian patent published in 2021 - IPC C12N15/864 

FIELD: gene therapy; molecular biology.

SUBSTANCE: the present invention relates to the isolated modified VP1 capsid protein of the adeno-associated virus of serotype 5 (AAV5), which contains one or more amino acid substitutions compared to the VP1 capsid protein of the wild-type AAV5, which increase the efficiency of transduction, as well as to the capsid and vector based on it. The isolated modified VP1 capsid protein of the adeno-associated virus of serotype 5 (AAV5) for high-efficient target cell transduction contains the amino acid sequence of the VP1 capsid protein of the AAV5 type encoded by the Cap gene, with one or more substitutions selected from the group: S651A, S2A and T711S, S2A, S651A and T711S. The isolated nucleic acid encodes a modified VP1 capsid protein of the adeno-associated virus of serotype 5 (AAV5). The isolated capsid for high-efficient target-cell transduction includes a modified VP1 capsid protein of the adeno-associated virus serotype 5 (AAV5). The isolated nucleic acid encodes a capsid that is used for high-efficient target-cell transduction. A vector based on recombinant adeno-associated virus of serotype 5 (rAAV5) for delivering a heterologous nucleic acid sequence to a subject includes: 1) capsid of any of the pp. 13-28 and 2) a heterologous nucleic acid sequence containing regulatory sequences that provide product expression. A pharmaceutical composition is used to deliver a gene product to a subject in need. The method for delivering the gene product to the subject in need involves injecting the subject with a vector based on rAAV5 or a pharmaceutical composition according to p. 35. A vector based on rAAV5 or a pharmaceutical composition is used to treat the disease in the subject in need. A method for producing a vector based on rAAV5 involves transfection of the producing cells with nucleic acid.

EFFECT: invention allows the optimization of vectors for maximum tissue transduction while minimizing the dose of the vector.

46 cl, 6 dwg, 4 ex